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

First listed

Feb 21, 2025

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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