Nationwide Children's Hospital
This is a Phase II open-label study to investigate the safety and efficacy of ACT001 in patients with DIPG and H3K27-altered HGG.
Patients will be enrolled on either Cohort A for newly diagnosed DIPG or on Cohort B \[ENROLLMENT PAUSED\] 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…
Inclusion Criteria: 1. Patients must be ≥ 12 months and ≤ 39 years of age at the time of study enrollment. 2. Diagnosis: * Cohort A: Newly Diagnosed DIPG * Patients with newly-diagnosed DIPG with typical MRI findings (tumors with a pontine epicenter and diffuse involvement of at least 2/3 of the pons) with or without biopsy and have completed radiation therapy (RT) within 28 to 35 calendar day prior to start of therapy. * Patients must have started RT \<42 calendar days from radiographic diagnosis (for non-biopsied DIPG patients only) or definitive surgery, whichever is later.…
PO BID at 875 mg/m2 for 28 days