Clinical Trials / Research Studies

DIPG/High Grade Glioma Relapse / Refractory

19-040: Study of the Bromodomain (BRD) and Extra-Terminal Domain (BET) Inhibitors BMS-986158 and BMS-986378 in Pediatric Cancer

• Ages: 1 Year to 21 Years  

Arm 1 of this research study is studying an investigational drug called BMS-986158 as a possible treatment for pediatric solid tumors or lymphoma. Arm 2 of this research study is studying an investigational drug called BMS-986378 (also known as CC-90010) as a possible treatment for pediatric brain t ...More

A Trial of Dabrafenib, Trametinib and Hydroxychloroquine for Patients With Recurrent LGG or HGG With a BRAF Aberration

• Ages: 1 year and ≤ 30 years old

This phase I/II trial studies the side effects and best dose of adding hydroxychloroquine to dabrafenib and/or trametinib, and to see how well they work in treating children with low grade or high grade brain tumors previously treated with similar drugs that did not respond completely (progressive) ...More

AMXT1501: 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

• Ages: 12 Years and older 
• Gender: Female, Male

The objective of this study is to determine the safety and tolerability of oral AMXT 1501 dicaprate (AMXT1501) in combination with IV DFMO in patients with advanced solid tumors, or DIPG/DMG. Secondary objectives include characterization of plasma pharmacokinetics (PK), pharmacodynamic (PD), and oth ...More

APEC1621: Pediatric MATCH: Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders

• Ages: 12 Months to 21 Years

PURPOSE: This screening and multi-sub-study phase II trial studies how well treatment that is directed by genetic testing works in pediatric patients with solid tumors, non-Hodgkin lymphomas, or histiocytic disorders that have progressed following at least one line of standard systemic therapy and/o ...More

ENV-ONC-101: A Study Evaluating the Safety and Efficacy of ENV-101 (Taladegib) in Patients With Advanced Solid Tumors Harboring PTCH1 Loss of Function Mutations

• Ages: 1 Year to 22 Years

This study employs a 2-stage design that aims to evaluate the efficacy and safety of ENV- 101, a potent Hedgehog (Hh) pathway inhibitor, in patients with refractory advanced solid tumors characterized by loss of function (LOF) mutations in the Patched-1 (PTCH1) gene. Stage 1 of this study will enrol ...More

GCC1949: Pediatric Trial of Indoximod With Chemotherapy and Radiation for Relapsed Brain Tumors or Newly Diagnosed DIPG

• Ages: 3 years to 21 years of age

Indoximod was developed to inhibit the IDO (indoleamine 2,3-dioxygenase) enzymatic pathway, which is important in the natural regulation of immune responses. This potent immune suppressive mechanism has been implicated in regulating immune responses in settings as diverse as infection, tissue/organ ...More

INCB7839 in Treating Children With Recurrent/Progressive High-Grade Gliomas

• Ages: 3 Years to 21 Years

This is a multicenter phase 1 trial of INCB7839 for children with recurrent or progressive high-grade gliomas, including, but not limited to, diffuse intrinsic pontine glioma (DIPG) and other diffuse midline gliomas (DMGs), after upfront therapy. INCB7839 is an inhibitor of the ADAM (A Disintegrin a ...More

LOXO-RET-18036: A Study of Oral LOXO-292 in Pediatric Patients With Advanced Solid or Primary Central Nervous System Tumors

• Ages: 6 Months to 21 Years  

To determine the safety of oral LOXO-292 in pediatric patients with advanced solid tumors: Dose limiting toxicities (DLTs) [ Time Frame: During the first 28-day cycle of LOXO-292 treatment ] LOXO-RET-18036: A Phase 1/2 Study of the Oral RET Inhibitor LOXO 292 in Pediatric Patients With Advanced RET ...More

PBTC-045: A Safety and Preliminary Efficacy trial of MK-3475 (pembrolizumab; anti-PD-1) in Children with recurrent, progressive or refractory high-grade gliomas (HGG) and DIPGs

• Ages: From 1 to 21 years old

This clinical trial studies the side effects and best dose of pembrolizumab and to see how well it works in treating younger patients with high-grade gliomas (brain tumors) or diffuse intrinsic pontine gliomas (brain stem tumors) that have come back, progressed, or have not responded to previous tre ...More

PBTC-048: Feasibility Trial of Optune for Children With Recurrent or Progressive Supratentorial High-Grade Glioma and Ependymoma

PURPOSE: This feasibility trial studies how well the Optune device works in treating younger patients with recurrent/refractory/progressive supratentorial malignant glioma and ependymoma. The TTFields generated by Optune device may inhibit the growth of tumor.&#x2028 DETAILED DESCRIPTION: Primary Ob ...More