New hope for kids with deadly brain tumors: ACT001 trial launches

NCT ID NCT06838676

Recruiting now Disease control Sponsor: Nationwide Children's Hospital Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 13, 2026 · Updated 2 times

Summary

This study tests an experimental drug called ACT001 in children and young adults (ages 1 to 39) with two aggressive brain tumors: DIPG and H3K27-altered high-grade gliomas. The goal is to see if ACT001 can improve survival or shrink tumors after radiation therapy. About 60 participants will be enrolled across two groups: newly diagnosed DIPG patients and those with progressive or recurrent disease.

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Conditions

The condition(s) this trial relates to.

diffuse intrinsic pontine glioma diffuse midline glioma, H3 K27-altered glioma recurrent uveitis, horse

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DIFFUSE INTRINSIC PONTINE GLIOMAS (DIPG) H3K27-ALTERED HIGH GRADE GLIOMA PROGRESSIVE DIPG RECURRENT DIPG REFRACTORY DIPG

Contacts and locations

Locations

C.S. Mott Children's Hospital
RECRUITING

Ann Arbor, Michigan, 48109, United States
Children's Hospital Colorado
RECRUITING

Aurora, Colorado, 80045, United States
Children's Hospital of Philidelphia
NOT_YET_RECRUITING

Philidelphia, Pennsylvania, 19104, United States
Children's National Medical Center
RECRUITING

Washington D.C., District of Columbia, 20010, United States
Cincinnati Children's Hospital
RECRUITING

Cincinnati, Ohio, 45229, United States
Duke University Medical Center
NOT_YET_RECRUITING

Durham, North Carolina, 27708, United States
Emory University/Children's Healthcare of Atlanta
NOT_YET_RECRUITING

Atlanta, Georgia, 30322, United States
Hopp Children's Cancer Center at NCT Heidelberg (KiTZ)
NOT_YET_RECRUITING

Heidelberg, Baden-Wurttemberg, 69120, Germany
Montreal Children's Hospital
NOT_YET_RECRUITING

Montreal, Quebec, H4A3J1, Canada
Nationwide Children's Hospital
RECRUITING

Columbus, Ohio, 43235, United States
Nicklaus Children's Hospital
NOT_YET_RECRUITING

Miami, Florida, 33155, United States
Perth Children's Hospital
NOT_YET_RECRUITING

Perth, Western Australia, 6000, Australia
Queensland Children's Hospital
NOT_YET_RECRUITING

South Brisbane, Queensland, 4101, Australia
Royal Children's Hospital
NOT_YET_RECRUITING

Melbourne, Victoria, 3052, Australia
Seattle Children's Hospital
RECRUITING

Seattle, Washington, 98105, United States
St. Louis Children's Hospital
NOT_YET_RECRUITING

St Louis, Missouri, 63110, United States
Starship Children's Hospital
NOT_YET_RECRUITING

Auckland, Grafton, 1023, New Zealand
Sydney Children's Hospital
NOT_YET_RECRUITING

Randwick, New South Wales, 2031, Australia
Texas Children's Hospital
RECRUITING

Houston, Texas, 77030, United States
The Hospital for Sick Children (SickKids)
RECRUITING

Toronto, Ontario, M5G1X8, Canada

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