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

Conditions

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

Eligibility Criteria

Sex

ALL

Age

12 Months - 39 Years

Healthy Volunteers

Not accepted

Interventions

• ACT001 — DRUG
  PO BID at 875 mg/m2 for 28 days

Study Details

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

Key Dates

Start date

Jul 10, 2025

Status verified

May 2026

Primary completion

Jul 31, 2028

Completion

Jul 31, 2035

Study Design

Enrollment

60 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Cohort A
  Patients with newly-diagnosed DIPG with typical MRI findings
• Experimental: Cohort B
  Patients with progressive/recurrent DIPG or H3K27-altered HGG OR refractory disease

Primary Outcome Measure

Overall Survival (OS) for newly diagnosed DIPG [ Time Frame: From date on treatment until date of death due to any cause or date of last follow-up, assessed up to 60 months ]

Central Contacts

• Kelsey H Troyer, PhD
  16147223284
  [email protected]

Locations (12)

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