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 must be ≥ 12 months and ≤ 39 years of age at the time of study enrollment.
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.
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.
Single group
ACT001
DrugPO BID at 875 mg/m2 for 28 days
Overall Survival (OS) for newly diagnosed DIPG
To assess the overall survival for newly-diagnosed patients with DIPG treated with RT followed by ACT001.
Objective Response Rate (ORR) in Progressive/Refractory/Recurrent HGG after frontline RT
To assess the rate of objective response rate (defined as partial response + complete response) in patients who have been treated with at least frontline focal RT and have progressive DIPG or progressive/recurrent/refractory H3K27-altered HGG who are treated with ACT001
Overall Survival in Progressive/Refractory/Recurrent DIPG and H3K27-altered DIPG
To estimate the overall survival and duration of disease control for patients who have been treated with at least RT and have progressive DIPG or progressive/recurrent/refractory H3K27-altered HGG who are treated with ACT001
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Nationwide Children's Hospital
Lead sponsor
Accendatech USA Inc.
Collaborator
