RecruitingPhase 2NCT06838676

ACT001 for the Treatment of Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas

A Phase II Trial of ACT001 in Children and Adolescents With Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas

Sponsor

Nationwide Children's Hospital

Enrollment

60 participants

Start Date

Jul 10, 2025

Study Type

INTERVENTIONAL

Conditions

Diffuse Intrinsic Pontine Gliomas (DIPG)Progressive DIPG Refractory DIPG Recurrent DIPG H3K27-altered High Grade Glioma

Summary

This is a Phase II open-label study to investigate the safety and efficacy of ACT001 in patients with DIPG and H3K27-altered HGG.

Eligibility

Min Age: 12 MonthsMax Age: 39 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a drug called ACT001 for children and young adults with a rare and aggressive brain tumor called DIPG (Diffuse Intrinsic Pontine Glioma) or similar tumors with a specific genetic mutation (H3K27-altered). These tumors are very difficult to treat, and this study is looking for new options after standard radiation therapy. **You may be eligible if...** - You are between 1 year old and 39 years old - You have been newly diagnosed with DIPG, or have a high-grade glioma with the H3K27 mutation - You have completed radiation therapy within the required time window - Your doctor has determined your general health is adequate for treatment **You may NOT be eligible if...** - You have received other anti-cancer treatments (beyond surgery and steroids) before this trial - Your organ function (liver, kidney, blood counts) is not within acceptable ranges - You are pregnant or breastfeeding - You have certain other serious health conditions that would make treatment unsafe Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGACT001

PO BID at 875 mg/m2 for 28 days

Locations(20)

Children's Hospital Colorado

Aurora, Colorado, United States

Children's National Medical Center

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

Nicklaus Children's Hospital

Miami, Florida, United States

Emory University/Children's Healthcare of Atlanta

Atlanta, Georgia, United States

C.S. Mott Children's Hospital

Ann Arbor, Michigan, United States

St. Louis Children's Hospital

St Louis, Missouri, United States

Duke University Medical Center

Durham, North Carolina, United States

Cincinnati Children's Hospital

Cincinnati, Ohio, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Children's Hospital of Philidelphia

Philidelphia, Pennsylvania, United States

Texas Children's Hospital

Houston, Texas, United States

Seattle Children's Hospital

Seattle, Washington, United States

Sydney Children's Hospital

Randwick, New South Wales, Australia

Queensland Children's Hospital

South Brisbane, Queensland, Australia

Royal Children's Hospital

Melbourne, Victoria, Australia

Perth Children's Hospital

Perth, Western Australia, Australia

The Hospital for Sick Children (SickKids)

Toronto, Ontario, Canada

Montreal Children's Hospital

Montreal, Quebec, Canada

Hopp Children's Cancer Center at NCT Heidelberg (KiTZ)

Heidelberg, Baden-Wurttemberg, Germany

Starship Children's Hospital

Auckland, Grafton, New Zealand

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

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NCT06838676