NCT06838676

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Nationwide Children's Hospital · Academic / Other
• Sex: All
• Age: 12 Months – 39 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Patients will be enrolled on either Cohort A for newly diagnosed DIPG or on Cohort B for those with progressive / refractory DIPG or progressive / recurrent / refractory H3K27-altered HGG. Each cohort will receive ACT001 at 875 mg/m2 orally BID for 28 days (1 cycle of treatment), up to a maximum dose of 1700 mg BID. If patients are experiencing clinical benefit from study therapy, they will continue to receive ACT001 in repeat 28-day cycles for up to 26 cycles (approximately 2 years) or until disease progression, whichever occurs first. Continuation of treatment beyond 26 cycles may be considered if patients are receiving clinical benefit from the study, at the discretion of the sponsor and treating physician.

Conditions

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

Interventions

Type	Name	Description
DRUG	ACT001	PO BID at 875 mg/m2 for 28 days

Timeline

• Start date: 2025-07-10
• Primary completion: 2028-07-01
• Completion: 2035-07-01
• First posted: 2025-02-21
• Last updated: 2026-03-27

Locations

20 sites across 5 countries: United States, Australia, Canada, Germany, New Zealand

Regulatory

• FDA-regulated drug study