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Clinical trialNCT03605550Active, not recruitingPhase 1Interventional

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

SponsorNationwide Children's HospitalEnrollment64Diffuse Intrinsic Pontine GliomaHigh Grade Glioma
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

10 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States
  • Children'S Hospital Colorado

    Aurora, Colorado, United States

  • Children'S National Medical Center

    Washington D.C., District of Columbia, United States

  • Ann & Robert H. Lurie Children'S Hospital of Chicago

    Chicago, Illinois, United States

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, United States

  • Duke University Medical Center

    Durham, North Carolina, United States

  • Cincinnati Children'S Hospital Medical Center

    Cincinnati, Ohio, United States

  • Nationwide Children'S Hospital

    Columbus, Ohio, United States

  • the Children'S Hospital of Philadelphia

    Philadelphia, Pennsylvania, United States

  • Texas Children'S Hospital

    Houston, Texas, United States

  • Seattle Children'S Hospital

    Seattle, Washington, United States

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

PTC596
Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start
1 Aug 2018
Primary completion
1 Mar 2024
Completion
1 Mar 2029
Last update posted
27 Mar 2026
Registered enrollment
64
Registry id
NCT03605550

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.