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| Diagnosis | Diffuse Intrinsic Pontine Gliomas (DIPG), H3K27-altered High Grade Glioma | Study Status | Open |
| Phase | II |
| Age | 12 Months to 39 Years | Randomisation | NO |
| Line of treatment | First line treatment, Disease relapse or progression |
| Routes of Treatment Administration | Drug: ACT001 (Oral, twice a day) |
| Last Posted Update | 2026-07-22 |
| ClinicalTrials.gov # | NCT06838676 |
International Sponsor
Nationwide Children's HospitalPrincipal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Anirban DasCentres
Study Description
This study is eligible for STEP-1 funding. Find more information here.
This research study will investigate how safe and effective a drug called ACT001 is in patients with either Diffuse Intrinsic Pontine Gliomas (DIPG) or High-Grade Gliomas (with a H3K27 genetic alteration.)
Inclusion Criteria
- Ages 12 months to 39 years old
- Cohort A: Newly diagnosed DIPG (Diffuse Intrinsic Pontine Gliomas)
- Cohort B: DIPG or H3K27-altered HGG (High Grade Glioma) that came back, got worse, or didn’t respond to other treatment
- Must be able to do at least half of normal daily activities
- Cohort B must be recovered from past treatment side effects and follow required waiting times after chemo, immunotherapy, or radiation
- Must have healthy bone marrow, kidneys, liver, and heart
- Must meet all bloodwork requirements
- Seizures must be controlled; neurologic problems must be stable
- Must be at least 3 months after autologous stem cell transplant; no allogeneic transplants allowed
Other inclusion and exclusion criteria may apply and will be discussed with you by the study team.
