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 at Norton Children’s Cancer Institute

Learn more about participating in a clinical trial.

Active clinical trials at Norton Children’s Cancer Institute and the UofL School of Medicine include:

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

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)

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)

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-Lymphobl

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

Central Nervous System Germ Cell Tumors (CNS GCT)

Phase 2 Trial of Response-Based Radiation Therapy for Patients with Localized Central Nervous System Germ Cell Tumors (CNS GCT)

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).

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

Heart Failure

Pharmacologic Reversal of Ventricular Remodeling in Childhood Cancer Survivors at Risk for Heart Failure (PREVENT-HF): A Phase 2b Randomized Placebo-Controlled (Carvedilol) Trial

Hepatic Malignancy

Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)

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)

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)

Langerhans Cell Histiocytosis

International Collaborative Treatment Protocol For Children And Adolescents With Langerhans Cell Histiocytosis

Leukemia

Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome

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

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)

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

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 CYC

Medulloblastoma

Phase II Trial of Eflornithine/DFMO as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma

Neuroblastoma

Utilizing Response and Biology Based Risk Factors to Guide Therapy in Patients with Non-High Risk Neuroblastoma

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 mainten

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))

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

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.

Solid Tumors

A5481092 PHASE 1/2 STUDY TO EVALUATE PALBOCICLIB(IBRANCE®) IN COMBINATION WITH IRINOTECAN AND TEMOZOLOMIDE AND/OR IN COMBINATION WITH TOPOTECANAND CYCLOPHOSPHAMIDE IN PEDIATRIC PATIENTS WITH RECURRENT OR REFRACTORY SOLID TUMORS

Solid Tumors

A Phase 1/2 Study of Lenvatinib in Combination With Everolimus in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors

Wilms Tumor

Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)

Cancer – 7725

Norton Children’s Cancer Institute

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