Children's Cancer Clinical Trials Repository

ANZCHOG coordinates some of the most important childhood cancer research across Australia and New Zealand

Our clinical trials registry brings together every active and upcoming trial in one place – giving families, clinicians, and researchers a clear picture of what’s available, where, and for whom.

Whether you’re exploring options for a child in your care or looking for a study site, this registry is your starting point.

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Complete
Phase 1

1367.1

An open label, Phase Ia/Ib dose finding study with BI 894999 orally administered once a day in patients with advanced malignancies with repeated administration in patients with clinical benefit
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
>18 years
N/A
Temporarily Closed to Recruitment
Phase 2

AALL15P1

A Groupwide Pilot Study to Test the Tolerability and Biologic Activity of the Addition of Azacitidine (NSC# 102816) to Chemotherapy in Infants With Acute Lymphoblastic Leukemia (ALL) and KMT2A (MLL) Gene Rearrangement
Leukaemia / Lymphoma / Blood Disorders
<1 year
N/A
Closed to Recruitment
Phase 3

AALL1631

International Phase 3 trial in Philadelphia chromosome-positive acute lymphoblastic leukaemia (Ph+ ALL) testing imatinib in combination with two different cytotoxic
1 year

|

21 years
N/A
Closed to Recruitment
Phase 3

AALL1731

A Phase 3 Trial Investigating Blinatumomab (IND# 117467& NSC# 765986) in Combination with Chemotherapy in Patients with Newly Diagnosed Standard Risk or Down Syndrome B Lymphoblastic Leukemia (B-ALL) and the Treatment of Patients with Localized B-Lymphoblastic Lymphoma (B-LLy)
Leukaemia / Lymphoma / Blood Disorders
1 year

|

31 years
N/A
Open
Phase 3

AALL1732

A Phase 3 Randomized Trial of Inotuzumab Ozogamicin (NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy
Leukaemia / Lymphoma / Blood Disorders
1 year

|

25 years
Queensland Children’s Hospital John Hunter Children’s Hospital Monash Health Clayton Royal Children’s Hospital Women’s and Children’s Hospital Perth Children’s Hospital Starship Children’s Hospital Christchurch Hospital
Open
Phase 2

AALL1821

Phase 2 Study of Blinatumomab (NSC# 765986, IND# 125462) in Combination with Nivolumab (NSC# 748726, IND# 125462), a Checkpoint Inhibitor of PD-1, in B-ALL Patients
Leukaemia / Lymphoma / Blood Disorders
1 year

|

31 years
Queensland Children’s Hospital John Hunter Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead
Preparing to Open
Phase 3

AALL2131

A Study Testing the Combination of Dasatinib or Imatinib to Chemotherapy Treatment With Blinatumomab for Children, Adolescents, and Young Adults With Philadelphia Chromosome Positive (Ph+) or ABL-Class Philadelphia Chromosome-Like (Ph-Like) B-cell Acute Lymphoblastic Leukemia (B-ALL)
Leukaemia / Lymphoma / Blood Disorders
>1 year

|

46 years
Queensland Children’s Hospital Perth Children’s Hospital
Open
Phase 3

AAML1531

Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome
Leukaemia / Lymphoma / Blood Disorders
91 days

|

3 years
Sydney Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead Christchurch Hospital
Open
Phase 3

AAML1831

A Phase 3 Randomized Trial for Patients With De Novo AML Comparing Standard Therapy Including Gemtuzumab Ozogamicin (GO) to CPX-351 With GO, and the Addition of the FLT3 Inhibitor Gilteritinib for Patients With FLT3 Mutations
Leukaemia / Lymphoma / Blood Disorders
<21 years
Queensland Children’s Hospital John Hunter Children’s Hospital
Closed to Recruitment
Phase 2

AAML18P1

Stopping Tyrosine Kinase Inhibitors (TKI) to Assess Treatment-Free Remission (TFR) in paediatric Chronic Myeloid Leukemia – Chronic Phase (CML-CP)
Leukaemia / Lymphoma / Blood Disorders
<25 years
N/A
Closed to Recruitment
Phase 2

ACNS1221

A Phase II Study for the Treatment of Non-Metastatic Nodular Desmoplastic Medulloblastoma In Children Less Than 4 Years of Age
Brain and Spinal (Central Nervous System) Tumours
<47 months
N/A
Open
Phase 2

ACNS1422

A phase 2 study of reduced therapy for newly diagnosed average-risk WNT-driven medulloblastoma patients
Brain and Spinal (Central Nervous System) Tumours
3 years

|

21 years
N/A
Closed to Recruitment
Phase 2

ACNS1721

A Phase 2 Study of Veliparib (ABT-888, IND# 139199) and Local Irradiation, Followed by Maintenance Veliparib and Temozolomide, in Patients with Newly Diagnosed High-Grade Glioma (HGG) without H3 K27M or BRAFV600E Mutations
Brain and Spinal (Central Nervous System) Tumours
3 years

|

25 years
N/A
Open
Phase 1/2

ACNS1821

A Phase 1/2 Trial of Selinexor (KPT-330) and Radiation Therapy in Newly-Diagnosed paediatric Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)
Brain and Spinal (Central Nervous System) Tumours
12 months

|

21 years
Queensland Children’s Hospital Sydney Children’s Hospital Royal Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead Starship Children’s Hospital Christchurch Hospital
Open
Phase 2

ACNS2021

A Phase 2 Trial of Chemotherapy Followed by Response-Based Whole Ventricular &Amp; Spinal Canal Irradiation (WVSCI) for Patients With Localized Non-Germinomatous Central Nervous System Germ Cell Tumor
Brain and Spinal (Central Nervous System) Tumours
3 years

|

29 years
Queensland Children’s Hospital Sydney Children’s Hospital Royal Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead Starship Children’s Hospital Christchurch Hospital
Open
Phase 2

ACNS2321

A Phase II Trial Evaluating Chemotherapy followed by Response-Based Reduced Radiation Therapy for Patient with Central Nervous System Germinomas
Brain and Spinal (Central Nervous System) Tumours
3 years

|

29 years
Queensland Children’s Hospital Sydney Children’s Hospital Royal Children’s Hospital Perth Children’s Hospital
Open

ACOR

Australian Cardio-Oncology Registry
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<35 years
John Hunter Children’s Hospital Monash Health Clayton Royal Children’s Hospital Women’s and Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead
Closed to Recruitment
Phase 1

ACT001-AU-002

A study to evaluate the safety and tolerability and pharmacokinetics of ACT001 in children with advanced brain and solid tumors
Brain and Spinal (Central Nervous System) Tumours
1 year

|

21 years
N/A
Open
Phase 3

AGCT1531

A Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk paediatric and Adult Patients with Germ Cell Tumors
Germ Cell Tumours
Various depending on risk group & consult protocol
Queensland Children’s Hospital John Hunter Children’s Hospital Sydney Children’s Hospital Monash Health Clayton Royal Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead Starship Children’s Hospital Christchurch Hospital
Temporarily Closed to Recruitment
Phase 3

AHEP1531

Paediatric hepatic malignancy international therapeutic trial (PHITT)
Digestive System Tumours
<30 years
N/A
Open
Phase 3

ALLELE (ATA129-EBV-302)

Multicenter, Open Label, Phase 3 Study of Tabelecleucel for Solid Organ or Allogeneic Hematopoietic Cell Transplant Subjects with Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease after Failure of Rituximab or Rituximab and Chemotherapy
Leukaemia / Lymphoma / Blood Disorders
No limit
Royal Children’s Hospital Children’s Hospital at Westmead
Temporarily Closed to Recruitment

ALTE03N1

Genetic Analysis in Identifying Late-Occurring Complications in Childhood Cancer Survivors
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<99 years
N/A
Open

ALTE07C1

Neuropsychological and Social and Emotional and Behavioral Outcomes in Children with Cancer
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
>1 month
Perth Children’s Hospital
Open

ALTE11C2

Health Effects after Anthracycline and Radiation Therapy (HEART): Dexrazoxane and Prevention of Anthracycline-related Cardiomyopathy
Leukaemia / Lymphoma / Blood Disorders, Soft Tissue and Bone Tumours (Sarcoma)
No limit
Perth Children’s Hospital
Closed to Recruitment

ALTE15N2

LEAHRN (Late Effects After High-Risk Neuroblastoma) Study
Neuroblastoma and Ganglioneuroblastoma
5 years

|

50 years
N/A
Open
Phase 1/2

AMGEN 20180257

A Phase 1/2 Open-label Study to Investigate the Safety, Efficacy, and Pharmacokinetics of Administration of Subcutaneous Blinatumomab for the Treatment of Adults and Adolescents with Relapsed or Refractory B cell Precursor Acute Lymphoblastic Leukemia (R/R B-ALL) and Minimal Residual Disease Positive (MRD+) B-ALL
Leukaemia / Lymphoma / Blood Disorders
>12 years
Queensland Children’s Hospital
Open
Phase 1

AMGEN 20200034

A phase 1b study to evaluate the safety, tolerability, pharmacokinetics and efficacy of xaluritamig in adult, adolescent and paediatric participants with relapsed or refractory Ewing sarcoma
Soft Tissue and Bone Tumours (Sarcoma)
>2 years
N/A
Preparing to Open
Phase 1/2

AMGEN 20220107

A phase 1/2 study of subcutaneous blinatumomab administration in children with R/R and MRD+ B-ALL
Leukaemia / Lymphoma / Blood Disorders
>28 days

|

<12 years
Perth Children’s Hospital
Complete
Phase 1/2

AMXT1501-102

A Phase 1B/2A Study of the Safety, Tolerability and Initial Efficacy of Oral AMXT 1501 Dicaprate and Intravenous Difluoromethylornithine (DFMO) in Patients with Cancer
Brain and Spinal (Central Nervous System) Tumours
>12 years
N/A
Closed to Recruitment

ANBL00B1

Biomarkers in Tumor Tissue Samples From Patients With Newly Diagnosed Neuroblastoma or Ganglioneuroblastoma
Neuroblastoma and Ganglioneuroblastoma
<30 years
N/A
Closed to Recruitment
Phase 3

ANBL1232

Utilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients with Non-High-Risk Neuroblastoma
Neuroblastoma and Ganglioneuroblastoma
<18 months
N/A
Open
Phase 2

ANBL1821

A Phase 2 Randomised Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) (IND# TBD) in Children with Relapsed& Refractory or Progressive Neuroblastoma
Neuroblastoma and Ganglioneuroblastoma
>1 year
Queensland Children’s Hospital Sydney Children’s Hospital Royal Children’s Hospital Women’s and Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead
Closed to Recruitment
Phase 2

ANBL19P1

Pilot Study of Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination with Irinotecan and Temozolomide in the Post-Consolidation Setting for High-Risk Neuroblastoma
Neuroblastoma and Ganglioneuroblastoma
<30 years
N/A
Open
Phase 3

ANBL2131

A Phase 3 Study of Dinutuximab Added to Intensive Multimodal Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma
Neuroblastoma and Ganglioneuroblastoma
<30 years
Queensland Children’s Hospital Sydney Children’s Hospital Monash Health Clayton Perth Children’s Hospital Starship Children’s Hospital
Closed to Recruitment
Phase 3

ANHL1931

A Randomized Phase 3 Trial of Nivolumab (NSC# 748726) in Combination With Chemo-Immunotherapy for the Treatment of Newly Diagnosed Primary Mediastinal B-Cell Lymphoma
Leukaemia / Lymphoma / Blood Disorders
>2 years
N/A
Open
Phase 2

ANHL2121

Phase 2 Study of Tovorafenib (DAY101) in Relapsed and Refractory Langerhans Cell Histiocytosis
Leukaemia / Lymphoma / Blood Disorders
189 days

|

22 years
Queensland Children’s Hospital Sydney Children’s Hospital Perth Children’s Hospital
Open
Phase 3

ANZUP 1302

Phase 3 Accelerated BEP Trial: A randomised phase 3 trial of accelerated versus standard BEP chemotherapy for patients with intermediate and poor-risk metastatic Germ Cell Tumours
Germ Cell Tumours
11 years

|

50 years
Queensland Children’s Hospital John Hunter Children’s Hospital Starship Children’s Hospital Christchurch Hospital
Open
Phase 3

AOST2031

A Phase 3 Randomized Controlled Trial Comparing Open vs Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma
Soft Tissue and Bone Tumours (Sarcoma)
<50 years
Monash Health Clayton Royal Children’s Hospital Perth Children’s Hospital Starship Children’s Hospital Christchurch Hospital
Open
Phase 2/3

AOST2032

A Feasibility and Randomized Phase 2/3 Study of the VEFGR2/MET Inhibitor Cabozantinib in Combination with Cytotoxic Chemotherapy for Newly Diagnosed Osteosarcoma
Soft Tissue and Bone Tumours (Sarcoma)
<40 years
Queensland Children’s Hospital Sydney Children’s Hospital Royal Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead Starship Children’s Hospital Christchurch Hospital
Open
Phase 3

APAL2020D

A randomized phase 3 trial of fludarabine/cytarabine/gemtuzumab ozogamicin with or without venetoclax in children with relapsed AML
Leukaemia / Lymphoma / Blood Disorders
29 days

|

21 years
Queensland Children’s Hospital Royal Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead Starship Children’s Hospital Christchurch Hospital
Open
Phase 1/2

APAL2020SC

paediatric Acute Leukemia (PedAL) Screening Trial – Developing New Therapies for Relapsed Leukemias
Leukaemia / Lymphoma / Blood Disorders
<22 years
Queensland Children’s Hospital Royal Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead Starship Children’s Hospital Christchurch Hospital
Open

APEC14B1

Project: EveryChild A Registry and Eligibility Screening and Biology and Outcome Study
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<25 years
Queensland Children’s Hospital John Hunter Children’s Hospital Sydney Children’s Hospital Monash Health Clayton Royal Children’s Hospital Women’s and Children’s Hospital Perth Children’s Hospital Royal Hobart Hospital Children’s Hospital at Westmead Starship Children’s Hospital Christchurch Hospital
Closed to Recruitment
Phase 2

APEC1621F

NCI-COG paediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 Subprotocol of Ensartinib in Patients With Tumors Harboring ALK or ROS1 Genomic Alterations
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
12 months

|

21 years
N/A
Closed to Recruitment
Phase 2

APEC1621K

NCI-COG paediatric MATCH (Molecular Analysis for Therapy Choice) – Phase 2 Subprotocol of AG-120 (Ivosidenib) in Patients With Tumors Harboring IDH1 Mutations
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
12 months

|

21 years
N/A
Open
Phase 2

APEC1621N

NCI-COG paediatric MATCH (Molecular Analysis for Therapy Choice) – Phase 2 Subprotocol of LOXO-292 in Patients With Tumors Harboring RET Gene Alterations
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
12 months

|

21 years
N/A
Closed to Recruitment
Phase 1/2

APG-115-US-002

A Phase Ib/II Study of APG-115 in Combination With Pembrolizumab in Patients With Unresectable or Metastatic Melanomas or Advanced Solid Tumors
Breast tumours, CNS tumours, Digestive system tumours, Endocrine tumours, Eye tumours, Female genital tumours, Genetic tumour syndromes, Germ cell tumours, Haematolymphoid disorders, Head and neck tumours, Peripheral neuroblastic tumours, Renal and male genital tumours, Skin tumours, Soft tissue and bone tumours, Thoracic tumours
>12 years
N/A
Closed to Recruitment
Phase 1/2

APG-115-US-002

A Phase Ib/II Study of APG-115 in Combination With Pembrolizumab in Patients With Unresectable or Metastatic Melanomas or Advanced Solid Tumors
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
>12 years
N/A
Open
Phase 2

ARAR2221

A Phase 2 Study Using Chemoimmunotherapy with Gemcitabine, Cisplatin and Nivolumab in Newly Diagnosed Nasopharyngeal Carcinoma (NPC)
Head and neck tumours
<21 years
Perth Children’s Hospital Starship Children’s Hospital Christchurch Hospital
Closed to Recruitment

AREN03B2

Renal Tumors Classification, Biology, and Banking Study
Kidney (Renal) Tumours
<21 years
N/A
Open
Phase 2

AREN1921

Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)
Kidney (Renal) Tumours
<30 years
Queensland Children’s Hospital Royal Children’s Hospital Women’s and Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead Starship Children’s Hospital Christchurch Hospital
Open
Phase 3

AREN2231

A Study Using Risk Factors to Determine Treatment for Children With Favorable Histology Wilms Tumors (FHWT)
Kidney (Renal) Tumours
<30 years
Queensland Children’s Hospital
Open
Phase 2

ARET2121

Intravitreal Melphalan for Intraocular Retinoblastoma
Eye Tumours
<18 years
Royal Children’s Hospital Perth Children’s Hospital
Complete
Phase 1

ARRAY-162-115

A Multicenter, Open-label Phase 1b Study of the Combination of Binimetinib and Encorafenib in Adolescent Patients with Unresectable or Metastatic BRAF V600-mutant Melanoma
Skin tumours
12 years

|

17 years
N/A
Closed to Recruitment
Phase 3

ARST1431

A randomized Phase III study of vincristine& dactinomycin& cyclophosphamide (VAC) alternating with vincristine and irinotecan (VI) versus VAC/VI plus temsirolimus (TEM) in patients with intermediate risk (IR) rhabdomyosarcoma (RMS)
Soft Tissue and Bone Tumours (Sarcoma)
<40 years
N/A
Open
Phase 2

ARST1921

A Safety, Pharmacokinetic and Efficacy Study of a y-Secretase Inhibitor, Nirogacestat (PF-03084014) in Children and Adolescents With Progressive, Surgically Unresectable Desmoid Tumors
Soft Tissue and Bone Tumours (Sarcoma)
1 year

|

18 years
Perth Children’s Hospital
Open
Phase 3

ARST2031

A Randomized Phase 3 Trial of Vinorelbine, Dactinomycin, and Cyclophosphamide (VINO-AC) Plus Maintenance Chemotherapy with Vinorelbine and Oral Cyclophosphamide (VINO-CPO) vs Vincristine, Dactinomycin and Cyclophosphamide (VAC) plus VINO-CPO Maintenance in Patients with High Risk Rhabdomyosarcoma (HR-RMS)
Soft Tissue and Bone Tumours (Sarcoma)
<50 years
Queensland Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead
Open
Phase 3

ARST2032

A Prospective Phase 3 Study of Patients With Newly Diagnosed Very Low-Risk and Low-Risk Fusion Negative Rhabdomyosarcoma
Soft Tissue and Bone Tumours (Sarcoma)
<21 years
Queensland Children’s Hospital Sydney Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead Starship Children’s Hospital Christchurch Hospital
Open
Phase 3

ASCT2031

A Multi-Center, Phase 3, Randomized Trial of Matched Unrelated Donor (MUD) Versus HLA-Haploidentical Related (Haplo) Myeloablative Hematopoietic Cell Transplantation for Children, Adolescents, and Young Adults (AYA) With Acute Leukemia or Myelodysplastic Syndrome (MDS)
Leukaemia / Lymphoma / Blood Disorders
6 months

|

21 years
Perth Children’s Hospital Children’s Hospital at Westmead
Closed to Recruitment

ASSET

Acute Lymphoblastic Leukaemia Subtype and Side-Effects from Treatment – Nationwide analysis of severe treatment-related toxicity in childhood leukaemia
Leukaemia / Lymphoma / Blood Disorders
<18 years
N/A
Complete
Phase 4

B0661037

A randomised and open-label and active controlled and safety and extrapolated efficacy study in paediatric subjects requiring anticoagulation for the treatment of a venous thromboembolic event (Phase IV) Apixaban
Leukaemia / Lymphoma / Blood Disorders
<17 years
N/A
Closed to Recruitment
Phase 2

BIOMEDE

Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication
Brain and Spinal (Central Nervous System) Tumours
6 months

|

25 years
N/A
Closed to Recruitment
Phase 1/2

BLU-285-3101

A Phase 1/2, Single-arm Study to Evaluate the Safety, Pharmacokinetics, and Antitumor Activity of Avapritinib in paediatric Patients with Solid Tumors Dependent on KIT or PDGFRA Signaling
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
2 years

|

17 years
N/A
Open

BuGenes01

Implementing pharmacogenetics in the busulfan dosing method for children undergoing hematopoietic stem-cell transplantation: a prospective, multicentric, randomized clinical trial
Leukaemia / Lymphoma / Blood Disorders
<18 years
N/A
Complete
Phase 1/2

CA045020 / NKTR-214

Phase 1/2 Study of Bempegaldesleukin in Combination With Nivolumab in Children, Adolescents, and Young Adults With Recurrent or Refractory Malignancies (PIVOT IO 020)
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<30 years
N/A
Complete
Phase 1/2

CA224-069

A Phase 1/2 Study of the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of Relatlimab Plus Nivolumab in paediatric and Young Adult Participants with Recurrent or Refractory Classical Hodgkin Lymphoma and Non-Hodgkin Lymphoma
Leukaemia / Lymphoma / Blood Disorders
<30 years
N/A
Open
Phase 2

CABL001I12202

A phase II, multicenter, open-label, single arm study to evaluate the safety and efficacy of asciminib in pediatric participants newly diagnosed or previously treated with Philadelphia positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) with or without known T315I mutation
Leukaemia / Lymphoma / Blood Disorders
1

|

18 years
Queensland Children’s Hospital
Preparing to Open
Phase 1/2

CABL001L12101

A multi-center, open-label, phase I/II study to evaluate the safety and efficacy of asciminib with chemotherapy followed by asciminib plus blinatumomab in pediatric, adolescent, and young adult participants with relapsed or refractory BCR::ABL1-positive (Philadelphia positive, Ph+) or BCR::ABL1-like (Ph-like) acute lymphoblastic leukemia (ALL)
Leukaemia / Lymphoma / Blood Disorders
1

|

17 years
N/A
Closed to Recruitment
Phase 2

CAMPFIRE- J1S-MC-JP04

A Randomized, Open-Label, Phase 2 Study Evaluating Abemaciclib in Combination With Irinotecan and Temozolomide in Participants With Relapsed or Refractory Ewing’s Sarcoma
Soft Tissue and Bone Tumours (Sarcoma)
1 year

|

39 years
N/A
Temporarily Closed to Recruitment
Phase 1

CARTELL

A Phase 1 Study of CD19 Specific Chimeric Antigen Receptor T-cell for Therapy of persistent and Relapsed B-cell leukaemia and Lymphoma Post Allogeneic Stem Cell Transplantation (The CARTELL Study)
Leukaemia / Lymphoma / Blood Disorders
No limit
N/A
Open
Phase 1

CARTpaedST

Developing genetically modified TCells for the treatment of paediatric solid tumours
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<18 years
Children’s Hospital at Westmead
Open
Phase 2

CBYL719F12202 EPIK-P4

A Phase II single arm study to assess the efficacy, safety and pharmacokinetics of alpelisib (BYL719) in paediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS)
Genetic tumour syndromes
2 years

|

100 years
Sydney Children’s Hospital Women’s and Children’s Hospital
Open
Phase 2/3

CBYL719P12201

A Two-stage Double-blind, Randomized, Placebo-controlled Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib in paediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.
Soft Tissue and Bone Tumours (Sarcoma)
<100 years
Queensland Children’s Hospital Sydney Children’s Hospital
Closed to Recruitment
Phase 3

CCTL019A2205B

Long Term Follow-Up of Patients Exposed to Lentiviral-Based CAR T-Cell Therapy
<100 years
N/A
Closed to Recruitment
Phase 2

CDRB436G2201

Phase II Open-label Global Study to Evaluate the Effect of Dabrafenib in Combination With Trametinib in Children and Adolescent Patients With BRAF V600 Mutation Positive Low Grade Glioma (LGG) or Relapsed or Refractory High Grade Glioma (HGG)
Brain and Spinal (Central Nervous System) Tumours
3 years

|

25 years
N/A
Closed to Recruitment
Phase 1/2

CFZ2008 / 20140106

Phase 1b/2 Study of Carfilzomib in Combination with Dexamethasone and Mitoxantrone and PEG-asparaginase& and Vincristine (UK R3 Induction Backbone) in Children with Relapsed or Refractory Acute Lymphoblastic Leukaemia
Leukaemia / Lymphoma / Blood Disorders
1 month

|

21 years
N/A
Closed to Recruitment
Phase 1/2

CINC424F12201 / REACH4

A Phase I/II Open-label and Single-arm and Multi-center Study of Ruxolitinib Added to Corticosteroids in paediatric Patients With Grade II-IV Acute Graft vs. Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation
Leukaemia / Lymphoma / Blood Disorders
28 days

|

17 years
N/A
Closed to Recruitment
Phase 1

CLEE011Q12101

Phase I/II multicenter study to assess efficacy and safety of ribociclib (LEE011) in combination with topotecan and temozolomide (TOTEM) in paediatric patients with relapsed or refractory neuroblastoma and other solid tumors
Brain and Spinal (Central Nervous System) Tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Soft Tissue and Bone Tumours (Sarcoma)
12 months

|

21 years
N/A
Open
Phase 1/2

CO43810

A phase i/ii, open-label, single-arm, two-part trial to evaluate safety, tolerability, pharmacokinetics, and anti-tumor activity of glofitamab in combination with chemoimmunotherapy in paediatric and young adult participants with relapsed/refractory mature b-cell non-hodgkin lymphoma
Leukaemia / Lymphoma / Blood Disorders
6 months

|

30 years
Queensland Children’s Hospital Perth Children’s Hospital
Open
Phase 1

D0816C00025

A Phase 1, Open-label, Parallel Group Study to Investigate Olaparib Safety andTolerability, Efficacy and Pharmacokinetics in Paediatric Patients with Solid Tumours
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<18 years
Queensland Children’s Hospital Monash Health Clayton Children’s Hospital at Westmead
Open
Phase 2

DAY101-001 (FIREFLY)

A Phase 2, Open-Label, Multicenter Study to Evaluate the Safety and Efficacy of the Oral Pan-RAF Inhibitor DAY101 in paediatric Patients With BRAF-Altered, Recurrent or Progressive Low-Grade Glioma
Brain and Spinal (Central Nervous System) Tumours
6 months

|

25 years
Queensland Children’s Hospital Sydney Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead
Closed to Recruitment
Phase 1

DCL-17-001

A Phase 1, Open-Label, Dose Escalation Study of CLR 131 in Children and Adolescents with Select Solid Tumors, Lymphoma, and Malignant Brain Tumors
Brain and Spinal (Central Nervous System) Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Soft Tissue and Bone Tumours (Sarcoma)
2 years

|

25 years
N/A
Closed to Recruitment

DIPG

International Diffuse Intrinsic Pontine Glioma (DIPG)/Diffuse Midline Glioma (DMG) Registry and Repository
Brain and Spinal (Central Nervous System) Tumours
No limit
N/A
Complete
Phase 2

E7080-G000-230 (OLIE)

A Multicenter, Open-label, Randomized Phase 2 Study to Compare the Efficacy and Safety of Lenvatinib in Combination with Ifosfamide and Etoposide versus Ifosfamide and Etoposide in Children, Adolescents and Young Adults with Relapsed or Refractory Osteosarcoma
Soft Tissue and Bone Tumours (Sarcoma)
2 years

|

25 years
N/A
Complete
Phase 1

ECREST

A Phase 1 Study of Intravenous EGFR-ErbituxEDVSMIT (EEDVSMit) in Children with Recurrent / Refractory Solid or Brain and Spinal (Central Nervous System) Tumours Expressing Epidermal Growth Factor Receptor (EGFR)
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
2 years

|

21 years
N/A
Open
Phase 3

EFC16295

A Phase 3 Open-label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated paediatric Patients <12 Years of Age With Severe Hemophilia A
Leukaemia / Lymphoma / Blood Disorders
<11 years
N/A
Complete
Phase 1

EZH102

A Phase I Study of the EZH2 Inhibitor Tazemetostat in paediatric Subjects with Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
6 months

|

17 years
N/A
Open
Phase 3

Firefly-2

A Phase 3, Randomized, International Multicenter Trial of DAY101 Monotherapy Versus Standard of Care Chemotherapy in Patients With paediatric Low-Grade Glioma Harboring an Activating RAF Alteration Requiring First-Line Systemic Therapy
Brain and Spinal (Central Nervous System) Tumours
<25 years
Queensland Children’s Hospital Sydney Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead Starship Children’s Hospital
Open
Phase 2

FORTE

A Phase 2 Master Protocol to assess the efficacy and safety of FORE8394, an inhibitor of BRAF Class 1 and Class 2 alterations, in participants with cancer harboring BRAF alterations
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
>10 years
Sydney Children’s Hospital
Open
Phase 4

HeadStart4

Newly Diagnosed Children (<10 y/o) With Medulloblastoma and Other CNS Embryonal Tumors Clinical and Molecular Risk-Tailored Intensive and Compressed Induction Chemotherapy Followed by Consolidation With Randomization to Either Single Cycle or to Three Tandem Cycles of Marrow-Ablative Chemotherapy With Autologous Hematopoietic Progenitor Cell Rescue
Brain and Spinal (Central Nervous System) Tumours
<10 years
Children’s Hospital at Westmead Christchurch Hospital
Open

HoTRODS

Post mortem tumour donation for Diffuse Intrinsic Pontine Glioma – developing DIPG cell cultures to test novel therapeutic strategies
Brain and Spinal (Central Nervous System) Tumours
No limits
John Hunter Children’s Hospital Sydney Children’s Hospital Perth Children’s Hospital Starship Children’s Hospital
Closed to Recruitment
Phase 1

I3Y-MC-JPCS

A Phase 1b/2 Study of Abemaciclib in Combination With Irinotecan and Temozolomide (Part A) and Abemaciclib in Combination With Temozolomide (Part B) in paediatric and Young Adult Patients With Relapsed/Refractory Solid Tumors and Abemaciclib in Combination With Dinutuximab, GM-CSF, Irinotecan, and Temozolomide in paediatric and Young Adult Patients With Relapsed/Refractory Neuroblastoma (Part C)
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<21 years
N/A
Open
Phase 2

I3Y-MC-JPEH

Randomized, Open-Label, Phase 2 Study Evaluating Abemaciclib in Combination With Temozolomide Compared to Temozolomide Monotherapy in Children and Young Adults With Newly Diagnosed High-Grade Glioma Following Radiotherapy
Brain and Spinal (Central Nervous System) Tumours
<20 years
Queensland Children’s Hospital Children’s Hospital at Westmead
Open
Phase 1/2

iMATRIX (GO42286)

A Phase I/II, Open-Label, Multicenter, Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in paediatric Patients With ALK Fusion-Positive Solid or CNS Tumors for Whom Prior Treatment Has Proven to be Ineffective or for Whom There is No Satisfactory Treatment Available
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<17 years
Queensland Children’s Hospital Sydney Children’s Hospital Royal Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead
Closed to Recruitment
Phase 1

INTACT-TAA

A clinical trial of donor-derived WT1 specific T cells for prevention of relapse in combination with multiple pathogen specific T cells for prevention of infection in patients undergoing allogeneic haemopoietic stem cell transplant for acute myeloid leukaemia
Leukaemia / Lymphoma / Blood Disorders
1 year

|

70 years
N/A
Closed to Recruitment
Phase 1

J10-MC-JZHD

A Phase 1 Study of Aurora Kinase A Inhibitor LY3295668 erbumine as a Single Agent and in Combination in Patients with Relapsed/Refractory Neuroblastoma
Neuroblastoma and Ganglioneuroblastoma
2 years

|

21 years
N/A
Closed to Recruitment
Phase 1/2

J1S-MC-JV01

CAMPFIRE: A Randomized, Open-Label Phase 1/2 Study Evaluating Ramucirumab in paediatric Patients and Young Adults With Relapsed, Recurrent, or Refractory Desmoplastic Small Round Cell Tumor
Soft Tissue and Bone Tumours (Sarcoma)
12 months

|

29 years
N/A
Closed to Recruitment
Phase 1/2

J1S-MC-JV02

Randomized, Open-Label Phase 1/2 Study Evaluating Ramucirumab in paediatric Patients and Young Adults With Relapsed, Recurrent, or Refractory Synovial Sarcoma
Soft Tissue and Bone Tumours (Sarcoma)
12 months

|

29 years
N/A
Open

Levi's CATCH

LEVERAGING CHIMERIC ANTIGEN RECEPTOR-EXPRESSING T CELLS FOR CHILDREN WITH DIFFUSE MIDLINE GLIOMA (LEVI’S-CATCH)
Brain and Spinal (Central Nervous System) Tumours
<21 years
Sydney Children’s Hospital
Open

LIBIO

Liquid biopsies for molecular monitoring of paediatric solid tumours
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<25 years
Children’s Hospital at Westmead
Open
Phase 1/2

LIGHTBEAM 01A MK-9999-01A

LIGHTBEAM-U01 Substudy 01A: A Phase 1/2 Substudy to Evaluate the Safety and Efficacy of Zilovertamab Vedotin in paediatric and Young Adult Participants With Hematologic Malignancies or Solid Tumors
Leukaemia / Lymphoma / Blood Disorders, Neuroblastoma and Ganglioneuroblastoma, Soft Tissue and Bone Tumours (Sarcoma)
6 months

|

25 years
Queensland Children’s Hospital Sydney Children’s Hospital Royal Children’s Hospital
Open
Phase 1/2

LIGHTBEAM 01C MK9999-01C

LIGHTBEAM-U01 Substudy 01C: A Phase 1/2 Substudy to Evaluate the Safety and Efficacy of Patritumab Deruta in paediatric Participants With Relapsed or Refractory Solid Tumors
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
1 months

|

17 years
Queensland Children’s Hospital Sydney Children’s Hospital Royal Children’s Hospital
Preparing to Open

LIGHTBEAM 01D

A Phase 1b/2 Substudy to Evaluate the Safety and Efficacy of Ifinatamab Deruxtecan in paediatric Participants With Relapsed or Refractory Solid Tumors
N/A
Phase 2

LIGHTBEAM-01E

LIGHTBEAM-U01 Substudy 01E: A Phase 2 Substudy to Evaluate the Safety and Efficacy of Belzutifan in Participants With Solid Tumors
N/A
Closed to Recruitment
Phase 1

LOXO-EXT-17005

A Phase 1/2 Study of the TRK Inhibitor LOXO-195 in Adult and paediatric Subjects With Previously Treated NTRK Fusion Cancers
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
>1 month
N/A
Closed to Recruitment
Phase 1/2

LOXO-RET-18036

A Phase I/II Study of the Oral RET inhibitor LOXO 292 in Paediatric patients with Advanced RET-Altered Solid or Primary Central Nervous System Tumours
Brain and Spinal (Central Nervous System) Tumours, Endocrine Tumours, Soft Tissue and Bone Tumours (Sarcoma)
6 months

|

21 years
N/A
Closed to Recruitment
Phase 1/2

LOXO-TRK-15003

A Phase 1/2 Study of the Oral TRK Inhibitor LOXO-101 in Pediatric Patients With Advanced Solid or Primary Central Nervous System Tumors
Breast tumours, CNS tumours, Digestive system tumours, Endocrine tumours, Eye tumours, Female genital tumours, Genetic tumour syndromes, Germ cell tumours, Haematolymphoid disorders, Head and neck tumours, Peripheral neuroblastic tumours, Renal and male genital tumours, Skin tumours, Soft tissue and bone tumours, Thoracic tumours
<21 years
Sydney Children’s Hospital Women’s and Children’s Hospital
Closed to Recruitment
Phase 1/2

LOXO-TRK-15003

A Phase 1/2 Study of the Oral TRK Inhibitor LOXO-101 in paediatric Patients With Advanced Solid or Primary Central Nervous System Tumors
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<21 years
N/A
Open
Phase 3

LTS16294

A Phase 3 Open-label, Multicenter Study of the Long-term Safety and Efficacy of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients With Severe Hemophilia A
Leukaemia / Lymphoma / Blood Disorders, Neuroblastoma and Ganglioneuroblastoma, Soft Tissue and Bone Tumours (Sarcoma)
No limits
Queensland Children’s Hospital
Closed to Recruitment
Phase 1

M13-833

A Phase I Study of the Safety and Pharmacokinetics of Venetoclax in Paediatric and Young Adult Patients with Relapsed or Refractory Malignancies
Leukaemia / Lymphoma / Blood Disorders, Brain and Spinal (Central Nervous System) Tumours
0 years

|

25 years
N/A
Open
Phase 1

M20-429

A Single Arm, Open-Label, Phase 1b Trial of Epcoritamab in paediatric Patients With Relapsed/Refractory Aggressive Mature B-cell Neoplasms
Leukaemia / Lymphoma / Blood Disorders
1 year

|

26 years
Perth Children’s Hospital Children’s Hospital at Westmead
Open
Phase 2/3

MINI

A prospective, multi randomised, blinded, two-arm parallel trial of Medicinal Cannabis products for symptom burden in children with advanced cancer
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
6 months

|

21 years
John Hunter Children’s Hospital Royal Children’s Hospital
Closed to Recruitment
Phase 1/2

MK-3475-051

A Phase I/II Study of Pembrolizumab (MK-3475) in Children with advanced melanoma or a PD-L1 positive advanced and relapsed or refractory solid tumor or lymphoma
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
6 months

|

17 years
N/A
Closed to Recruitment
Phase 2

MK-7902-013

An Open-Label, Multicenter Phase 2 Basket Study to Evaluate the Antitumor Activity and Safety of Lenvatinib in Children, Adolescents, and Young Adults with Relapsed or Refractory Solid Malignancies
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
2 years

|

21 years
N/A
Closed to Recruitment
Phase 2

MK-8228-030

A Phase 2b open-label & single-arm study to evaluate pharmacokinetics& efficacy& safety and tolerability of letermovir in paediatric participants from birth to <18 years of age at risk of developing CMV infection and/or disease following allogeneic HSCT
Leukaemia / Lymphoma / Blood Disorders
<17 years
N/A
Closed to Recruitment
Phase 1

N2012-01

NANT 2012-01 Phase 1 study of Difluoromethylornithine (DFMO) and celecoxib with cyclophosphamide/topotecan for patients with relapsed or refractory neuroblastoma
Neuroblastoma and Ganglioneuroblastoma
2 years

|

30 years
N/A
Closed to Recruitment

ON TRK

PrOspective Non-interventional Study in Patients With Locally Advanced or Metastatic TRK Fusion Cancer Treated With Larotrectinib
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
No limit
N/A
Open
Phase 3

ONC201-108

ONC201 for the Treatment of Newly Diagnosed H3 K27M-mutant Diffuse Glioma Following Completion of Radiotherapy: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study
Brain and Spinal (Central Nervous System) Tumours
No limit
Queensland Children’s Hospital Sydney Children’s Hospital Perth Children’s Hospital Royal Hobart Hospital
Closed to Recruitment
Phase 3

P-105-202

Phase 2/3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of ALVR105 (Viralym-M) Compared to Placebo for the Prevention of AdV, BKV, CMV, EBV, HHV-6, and JCV Infection and/or Disease, in High-Risk Patients After Allogeneic Hematopoietic Cell Transplant.
Leukaemia / Lymphoma / Blood Disorders
No limit
N/A
Open
Phase 1

P305

Phase I clinical trial of adoptive transfer of multi-virus-specific T cells into TCRaß+/CD19+-depleted haploidentical HSCT recipients
Leukaemia / Lymphoma / Blood Disorders
3 months

|

18 years
John Hunter Children’s Hospital Royal Children’s Hospital
Closed to Recruitment
Phase 1/2

PCYC-1146-IM

Phase 1/2 Dose Finding and Safety and Efficacy Study of Ibrutinib in paediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)
Leukaemia / Lymphoma / Blood Disorders
<21 years
N/A
Closed to Recruitment
Phase 1/2

PEPN2121

A Phase 1/2 Study of Tiragolumab and Atezolizumab in Patients with Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
>12 months
N/A
Closed to Recruitment

PEPN22P1

A Pharmacokinetic Study of Vincristine in Infants Dosed According to BSA Banded Infant Dosing Tables and Older Children Dosed by Traditional BSA Methods
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<12 years
N/A
Closed to Recruitment
Phase 2

PersoMed-I

Personalized Risk-Adapted Therapy in Post-Pubertal Patients With Newly-Diagnosed Medulloblastoma
Brain and Spinal (Central Nervous System) Tumours
<15 years
N/A
Closed to Recruitment
Phase 1/2

Ponatinib-1501 /AALL1922

A Pivotal Phase 1/2, Single-Arm, Open-label Study to Evaluate the Safety and Efficacy of Ponatinib with Chemotherapy in paediatric Patients with Philadelphia Chromosome-Positive (Ph+) Acute Lymphoblastic Leukemia (ALL) Who Have Relapsed or Are Resistant or Intolerant to a Prior Tyrosine Kinase Inhibitor-Containing Therapy, or Who Have the T315I Mutation
Leukaemia / Lymphoma / Blood Disorders
1 year

|

21 years
N/A
Open

PPB DICER

International Pleuropulmonary Blastoma Registry for PPB, DICER1 and Associated Conditions
Brain and Spinal (Central Nervous System) Tumours, Genetic tumour syndromes, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<100 years
John Hunter Children’s Hospital Women’s and Children’s Hospital Perth Children’s Hospital Starship Children’s Hospital
Closed to Recruitment

PREDICT

Assessment of the Utility of Family-based (Trio) Whole-genome Sequencing for Cancer Predisposition Testing in Sequential Newly Diagnosed Paediatric and Adolescent Cancer Patients
Genetic tumour syndromes
<21 years
N/A
Closed to Recruitment
Phase 3

PRN1008-018

Ph3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study With Open Label Extension to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
Leukaemia / Lymphoma / Blood Disorders
>10 years
N/A
Preparing to Open
Phase 1/2

R3767-ONC-2320

A Phase 1/2 Open-Label, Safety and Efficacy Study of Neoadjuvant Fianlimab (Anti-LAG-3 Antibody) in Combination With Cemiplimab (Anti-PD-1 Antibody) and Cemiplimab Alone Followed by Adjuvant Fianlimab in Combination With Cemiplimab in Pediatric and Young Adult Participants With Recurrent or Progressive High-Grade Glioma or Pediatric and Adult Participants With Recurrent or Progressive Posterior Fossa-A Ependymoma
Brain and Spinal (Central Nervous System) Tumours
< 39 years
N/A
Open
Phase 1

R3ACT Fungal

A phase 1 trial of HLA-DR matched third party donor-derived fungus-specific cytotoxic T-lymphocytes in patients with invasive fungal disease post-allogeneic stem cell transplantation
Leukaemia / Lymphoma / Blood Disorders
1 year

|

80 years
Perth Children’s Hospital Children’s Hospital at Westmead
Complete
Phase 3

REACH2

A Phase III Randomized Open-Label Multi-centre Study of Ruxolitinib versus best available therapy in patients with Corticosteroid-refractory acute graft vs. host disease after allogeneic stem cell transplantation
Leukaemia / Lymphoma / Blood Disorders
>12 years
N/A
Open
Phase 2

RLY-2608-201

A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation
Genetic tumour syndromes
>2 years
Sydney Children’s Hospital
Open
Phase 2

S095032

A Phase 2, single arm, open-label study of vorasidenib in pediatric participants aged 12 to < 18 years old with Grade 2 astrocytoma or oligodendroglioma with an IDH1 or IDH2 mutation
CNS tumours
12 years

|

17 years
Sydney Children’s Hospital
Closed to Recruitment

SARS-CoV-2

A multicentre study of SARS-CoV-2 infection in children with cancer, immunodeficiency or following stem cell transplant
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<21 years
N/A
Open

SCRIPT

Using single-cell RNA-sequencing to identify clonal enrichment in paediatric solid tumours
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<18 years
Sydney Children’s Hospital
Closed to Recruitment

SerOzNET

SARS-CoV-2 post-vaccine surveillance studies in Australian children and adults with cancer
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
>12 years
N/A
Closed to Recruitment
Phase 2

SJMB12

A Clinical and Molecular Risk-Directed Therapy For Newly Diagnosed Medulloblastoma
Brain and Spinal (Central Nervous System) Tumours
3 years

|

39 years
N/A
Closed to Recruitment
Phase 1/2

T2016-002

A Phase I/II Study of Nivolumab in Combination with 5-azacytidine in paediatric patients with relapsed/refractory acute myeloid leukemia
Leukaemia / Lymphoma / Blood Disorders
1 year

|

30 years
N/A
Closed to Recruitment
Phase 1/2

T2017-002

A TACL Phase 1/2 Study of PO Ixazomib in combination with chemotherapy for childhood relapsed or refractory ALL and Lymphoblastic Lymphoma (Takeda)
Leukaemia / Lymphoma / Blood Disorders
1 year

|

21 years
N/A
Open

T2018-001

A retrospective cohort study of response and survival in paediatric patients with relapsed and refractory acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LLy) treated with contemporary salvage therapy
Leukaemia / Lymphoma / Blood Disorders
<21 years
Royal Children’s Hospital Children’s Hospital at Westmead
Open
Phase 1

T2020-006

A Phase I Study of Tagraxofusp With or Without Chemotherapy in paediatric Patients with Relapsed or Refractory CD123 Expressing Hematologic Malignancies
Leukaemia / Lymphoma / Blood Disorders
1 year

|

21 years
Children’s Hospital at Westmead
Closed to Recruitment

TAILOR

Safety and oversight of the individually tailored treatment approach: A novel pilot study
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
>2 year
N/A
Open
Phase 3

TAK-620-2004

A Phase 3, Open-label, Single-arm, Repeated-dose Study to Evaluate the Safety and Tolerability, Pharmacokinetics, and Antiviral Activity of Maribavir for the Treatment of Cytomegalovirus (CMV) Infection in Children and Adolescents Who Have Received a Hematopoietic Stem Cell Transplant (HSCT) or a Solid Organ Transplant (SOT)
Leukaemia / Lymphoma / Blood Disorders
<17 years
Queensland Children’s Hospital Sydney Children’s Hospital Royal Children’s Hospital Perth Children’s Hospital
Closed to Recruitment
Phase 2

TAPISTRY

Tumor-Agnostic Precision Immunooncology and Somatic Targeting Rational for You (TAPISTRY) Phase II Platform Trial
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
No limit
N/A
Open

The CLOCK Trial

Preventing adverse events during paediatric cancer treatment: A multi-site hybrid randomised controlled trial of catheter lock solutions (The CLOCK trial)
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<18 years
Queensland Children’s Hospital Monash Health Clayton Royal Children’s Hospital Starship Children’s Hospital
Closed to Recruitment
Phase 2/3

TOTAL 17

Total Therapy XVII for Newly Diagnosed Patients With Acute Lymphoblastic Leukemia and Lymphoma
Leukaemia / Lymphoma / Blood Disorders
1 year

|

18 years
N/A
Open
Phase 1/2

TPX-0005-07

A phase 1/2, open-label safety, tolerability, pharmacokinetics and anti-tumour activity study of repotrectinib in paediatric and young adult subjects with advanced or metastatic malignancies harbouring ALK, ROS1 or NTRK1-3 alterations
, Brain and Spinal (Central Nervous System) Tumours, Digestive System Tumours, Endocrine Tumours, Eye Tumours, Female genital tumours, Genetic tumour syndromes, Germ Cell Tumours, Leukaemia / Lymphoma / Blood Disorders, Head and neck tumours, Neuroblastoma and Ganglioneuroblastoma, Kidney (Renal) Tumours, Skin tumours, Soft Tissue and Bone Tumours (Sarcoma), Chest and Lung (Thoracic) Tumours
<25 years
Queensland Children’s Hospital Sydney Children’s Hospital Perth Children’s Hospital Children’s Hospital at Westmead

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