RecruitingPhase 2

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

United States60 participantsStarted 2025-07-10

Plain-language 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.

Age range12 Months – 39 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Patients must be ≥ 12 months and ≤ 39 years of age at the time of study enrollment.
✓. Diagnosis:
✓. Disease Status
✓. Performance Level: Karnofsky Performance Scale score ≥ 50% for patients \> 16 years of age and Lansky Performance Scale score \> 50% for patients ≤ 16 years of age (applies to all patients) Note: Patients who are unable to walk because of paralysis, but who are capable of using a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
✓. Prior anti-cancer therapy:

✕. Myelosuppressive chemotherapy: At least 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea).
✕. Hematopoietic growth factors: At least 14 days after the last dose of a long-acting growth factor (e.g. Neulasta) or 7 days for short-acting growth factor.
✕. Biologic (anti-neoplastic agent): At least 7 days after the last dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair.
✕. Immunotherapy: At least 42 days after the completion of any type of immunotherapy, e.g. tumor vaccines.
✕. Monoclonal antibodies: \> 21 days must have elapsed from the infusion of last dose of antibody and toxicity related to antibody therapy must be recovered to Grade ≤ 1

Overall Survival (OS) for newly diagnosed DIPG

Timeframe: From date on treatment until date of death due to any cause or date of last follow-up, assessed up to 60 months

Objective Response Rate (ORR) in Progressive/Refractory/Recurrent HGG after frontline RT

Timeframe: Date on treatment through 30 days following end of protocol treatment

NCT IDNCT06838676

SponsorNationwide Children's Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2028-07

Kelsey H Troyer, PhD

Who can participate