Childhood Cancer Clinical Trials and Research

Norton Children’s Cancer Institute, affiliated with the UofL School of Medicine, actively participates in pediatric oncology clinical trials and research. Norton Children’s Cancer Institute is a long-standing member of the Children’s Oncology Group, the world’s largest group of hospitals treating children with cancer. These hospitals follow the same best practices, protocols and therapy regimens for childhood cancer treatment. This means children from across the country have access to the same clinical trials offering the latest treatments while working to decrease possible long-term effects.

Norton Children’s Cancer Institute also is part of the Beat Childhood Cancer research consortium. The consortium is a group of 40-plus universities and children’s hospitals offering a worldwide network of childhood cancer research trials coordinated by Helen DeVos Children’s Hospital in Grand Rapids, Michigan.

These trials are based on the work of a group of researchers connected with laboratory programs that develop new therapies for high-risk pediatric cancers — all with the goal of finding a cure.

Open trials available via Beat Childhood Cancer

Active Clinical Trials and Studies at Norton Children’s Cancer Institute and the UofL School of Medicine

Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

A Randomized Trial of a Mobile Health and Social Media Physical Activity Intervention among Adolescent and Young Adult Childhood Cancer Survivors
Study Type: Observational
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: COG

Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic L

A Randomized Web-based Physical Activity Intervention among Children and Adolescents with Acute Lymphoblastic Leukemia
Study Type: Registry
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic L

An Open-Label, Multicenter Study of RC-P in Patients with Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic Lymphoma (LBL) Following Hypersensitivity to E. coli-derived Asparaginases
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Jazz Pharmaceuticals

Acute Myeloid Leukemia (AML)

A Phase 1/2 Study of CPX-351 (NSC# 775341; IND #129443) Alone Followed by Fludarabine, Cytarabine, and G-CSF (FLAG) for Children with Relapsed Acute Myeloid Leukemia (AML)
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

B-Acute Lymphoblastic Leukemia (B-ALL)

A Phase 2 Study of Inotuzumab Ozogamicin (NSC# 772518, IND#133494) in Children and Young Adults with Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL)
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

B-Acute Lymphoblastic Leukemia (B-ALL)

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)
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

B-Acute Lymphoblastic Leukemia (B-ALL)

AALL1732: A Phase 3 Randomized Trial of Inotuzumab Ozogamicin (IND#:133494, 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
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Behavior

Neuropsychological, Social, Emotional, and Behavioral Outcomes in Children with Cancer
Study Type: Observational
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Cancer

Renal tumors classifcation, biology and banking study.
Study Type: Specimen Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Cancer

COG ABTR04B1 ESTABLISHING CONTINUOUS CELL LINES AND XENOGRAFTS FROM PEDIATRIC CANCERS FOR BIOLOGICAL AND PRECLINICAL THERAPEUTIC STUDIES
Study Type: Specimen Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Cancer

Long-Term Follow-Up of Patients Previously Entered on Children's Cancer Group Clinical Trials (see below for list of studies)
Study Type: Observational
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Chronic Myeloid Leukemia (CML)

AAML1921-ITCC-054: A Phase I/II study of Bosutinib in pediatric patients with newly diagnosed chronic phase or resistant/intolerant Ph+ Chronic Myeloid Leukemia
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Classical Hodgkin Lymphoma (cHL)

An Open-label, Uncontrolled, Multicenter Phase II Trial of MK-3475 (Pembrolizumab) in Children and Young Adults with Newly Diagnosed Classical Hodgkin Lymphoma with Inadequate (Slow Early) Response to Frontline Chemotherapy (KEYNOTE 667).
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Merck Sharp & Dohme CORP

Education

Key adverse events following childhood cancer.
Study Type: Specimen Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Genomics

Adolescent and Family Perspectives on Genome-Informed Pediatric Cancer Therapy
Study Type: Observational
Esther Elise Knapp, M.D.
Esther Elise Knapp, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Curing Kids Cancer

Genomics

NCI-COG PEDIATRIC MATCH (MOLECULAR ANALYSIS FOR THERAPY CHOICE) MASTER VERSION CONTROL PROTOCOL
Study Type: Registry
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Germ Cell Tumors

AGCT1531 A Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients with Germ Cell Tumors
Study Type: Observational
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Germ Cell Tumors

A randomised phase 3 trial of accelerated versus standard BEP chemotherapy for patients with intermediate and poor-risk metastatic germ cell tumours
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Hematopoietic Stem Cell Donors

Identifying Predictors of Poor Health-Related Quality-of-Life among Pediatric Hematopoietic Stem Cell Donors
Study Type: Observational
William Tse M.D.
William Tse M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Pediatric Blood and Marrow Transplant Consortium (

Hepatic Malignancy

Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

High-Grade Glioma

A Phase 2 Study of Dabrafenib (NSC# 763760) with Trametinib (NSC# 763093) after Local Irradiation in Newly Diagnosed BRAFV600-Mutant High-Grade Glioma (HGG) (IND #145355)
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Hodgkin Lymphoma

A Phase III, Randomized Study of Nivolumab (Opdivo) or Brentuximab Vedotin (Adcetris) Plus AVD in Patients (Age >/= 12 Years) With Newly Diagnosed Advanced Stage Classical Hodgkin Lymphoma
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: COG

Intermediate Risk (IR) Rhabdomyosarcoma (RMS)

COG ARST1431 A Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating with Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864, IND# 122782) in Patients with Intermediate Risk (IR) Rhabdomyosarcoma (RMS)
Study Type: Drug Study
Kerry K. McGowan, M.D.
Kerry K. McGowan, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Langerhans Cell Histiocytosis

International Collaborative Treatment Protocol For Children And Adolescents With Langerhans Cell Histiocytosis
Study Type: Interventional
Kerry K. McGowan, M.D.
Kerry K. McGowan, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: North American Consortium for Histiocytosis

Leukemia

A Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib With Chemotherapy in Children With De Novo High-Risk CRLF2 Rearranged and/or JAK Pathway–Mutant Acute Lymphoblastic Leukemia
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Incyte Corporation

Low-Grade Glioma (LGG)

A Phase 3 Randomized Study of Selumetinib (IND # 77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Low-Grade Glioma (LGG)

A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Medulloblastoma

NEWLY DIAGNOSED CHILDREN (LESS THAN 10 YEARS OLD) WITH MEDULLOBLASTOMA AND OTHER CENTRAL NERVOUS SYSTEM EMBRYONAL TUMORS: CLINICAL AND MOLECULAR RISK-TAILORED INTENSIVE AND COMPRESSED INDUCTION CHEMOTHERAPY FOLLOWED BY CONSOLIDATION WITH EITHER SINGLE CYCLE (LOW RISK PATIENTS) OR RANDOMIZATION (HIGH RISK PATIENTS) TO either SINGLE-CYCLE or TO THREE TANDEM CYCLES OF MARROW-ABLATIVE CHEMOTHERAPY WITH AUTOLOGOUS HEMATOPOIETIC PROGENITOR CELL RESCUE
Study Type: Drug Study
Michael Huang, M.D.
Michael Huang, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Nationwide Children's Hospital

Medulloblastoma

Phase II Trial of Eflornithine/DFMO as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma
Study Type: Drug Study
Michael Huang, M.D.
Michael Huang, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Beat Childhood Cancer

Medulloblastoma

A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: COG

Neuroblastoma

Utilizing Response and Biology Based Risk Factors to Guide Therapy in Patients with Non-High Risk Neuroblastoma
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Neuroblastoma

LEAHRN (Late Effects After High-Risk Neuroblastoma) Study
Study Type: Specimen Study
Kerry K. McGowan, M.D.
Kerry K. McGowan, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Neuroblastoma

PEDS-PLAN – Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (A Study Using Molecular Guided Therapy with Induction Chemotherapy followed by a Randomized Controlled Trial of standard immunotherapy with or without DFMO followed by DFMO maintenance for Subjects with Newly Diagnosed High-Risk Neuroblastoma)
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Beat Childhood Cancer

Neuroblastoma

A Phase II Preventative Trial of DFMO as a Single Agent in Patients with High Risk Neuroblastoma in Remission (NMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO))
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Beat Childhood Cancer

Neuroblastoma

A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) (IND# 141913) in Children with Relapsed, Refractory or Progressive Neuroblastoma
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Neuroblastoma

A Pilot Study of Dinutuximab in Combination with Irinotecan and Temozolomide in the PostConsolidation Setting for High-Risk Neuroblastoma
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: COG

Neuroblastoma

Neuroblastoma Biology Study
Study Type: Specimen Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Philadelphia Chromosome Ph+ALL

International Phase 3 trial in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL) testing imatinib in combination with two different cytotoxic chemotherapy backbones.
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

reCURRENT OR Progressive Low-grade gliomas

A Phase 3 Study of Selumetinib (NSC# 748727, IND# 77782) or Selumetinib in Combination with Vinblastine for non-NF1, non-TSC Patients with Recurrent or Progressive Low-Grade Gliomas (LGGs) Lacking BRAFV600E or IDH1 Mutations
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: COG

Screening

COG APEC14B1 - Project Every Child: A Registry, Eligibility Screening, Biology and Outcome Study
Study Type: Specimen Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)

Sickle Cell Disease (SCD)

A Comparative Effectiveness Study of Peer Mentoring [PM] versus Structured Transition Education Based Intervention [STE] for the Management of Care Transitions in Emerging Adults with Sickle Cell Disease (SCD)
Study Type: Observational
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: PCORI

Sickle Cell Disease (SCD)

A Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched and HLA-Matched Bone Marrow for Patients with Sickle Cell Disease and Other Hemoglobinopathies
Study Type: Specimen Study
William Tse M.D.
William Tse M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Vanderbilt-Ingram Cancer Center

Solid Tumors

A5481092 PHASE 1/2 STUDY TO EVALUATE PALBOCICLIB(IBRANCE®) IN COMBINATION WITH IRINOTECAN AND TEMOZOLOMIDE AND/OR IN COMBINATION WITH TOPOTECAN AND CYCLOPHOSPHAMIDE IN PEDIATRIC PATIENTS WITH RECURRENT OR REFRACTORY SOLID TUMORS
Study Type: Drug Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Pfizer

Transient Myeloproliferative Disorder (TMD)

Biology Study of Transient Myeloproliferative Disorder (TMD) in Children with Down Syndrome (DS)
Study Type: Specimen Study
Ashok Raj, M.D.
Ashok Raj, M.D.
Principal Investigator
Norton Children's Cancer Institute,
affiliated with the UofL School of Medicine
Sponsor: Children's Oncology Group (COG)
Cancer – 7725

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Early symptoms of neuroblastoma can mimic other common childhood illnesses

Neuroblastoma is a rare type of childhood cancer that starts in the nerve cells of the sympathetic nervous system, and usually affects infants and children under age 5. The average age of diagnosis is 18 [...]

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Children with cyclic neutropenia are at higher risk of developing severe bacterial infections

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Boy fighting cancer has passion for giving back

Methotrexate and vincristine are complex words for most, but 7-year-old Beckham Goodale knows them and can pronounce them just as well as any doctor. They’re words no child should know, but this is the reality [...]

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A 39-year career of discovery as kids with cancer and blood disorders were able to thrive

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Early symptoms of neuroblastoma can mimic other common childhood illnesses

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Children with cyclic neutropenia are at higher risk of developing severe bacterial infections

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