MedTrace Logo

A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma

NCT03605550 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 64

Last updated 2026-03-27

No results posted yet for this study

Summary

The goal of this study is to evaluate the safety of the study drug PTC596 (Unesbulin) taken in combination with radiotherapy (RT) when given to pediatric patients newly diagnosed with High-Grade Glioma (HGG) including diffuse intrinsic pontine glioma (DIPG).

The main aims of the study are to:

* Find the safe dose of the study drug PTC596that can be given without causing serious side effects.
* Find out the amount of drug that enters blood (in all patients) and tumor (in patients who receive drug prior to a planned surgery for removal of their brain tumor)

During the first cycle (6-7weeks), patients will receive drug orally twice a week in combination with daily RT. During subsequent cycles (4 weeks each), they will receive only the study drug orally twice a week.

Funding Source - FDA OOPD

Conditions

Interventions

DRUG

PTC596

Oral tablets

RADIATION

Radiotherapy

Cycle1

Sponsors & Collaborators

  • PTC Therapeutics

    collaborator INDUSTRY

  • Nationwide Children's Hospital

    lead OTHER

Principal Investigators

  • Maryam Fouladi, MD · Nationwide Children's Hospital

  • Patricia Baxter, MD · Baylor College of Medicine

  • Margot Lazow, MD · Nationwide Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Months
Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-08-01
Primary Completion
2024-03-01
Completion
2029-03-01
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03605550 on ClinicalTrials.gov