NCT06838676 · Nationwide Children's Hospital
ACT001 for the Treatment of Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas
What this study is about
This is a Phase II where both patients and doctors know the treatment given study to investigate the safety and effectiveness of ACT001 in patients with DIPG and H3K27-altered HGG.
View original scientific description
This is a Phase II open-label study to investigate the safety and efficacy of ACT001 in patients with DIPG and H3K27-altered HGG.
Interventions
DRUG
ACT001
PO BID at 875 mg/m2 for 28 days
Primary outcome measures
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
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
Time frame: Date on treatment through 30 days following end of protocol treatment
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
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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 of radiographic diagnosis (for non-biopsied DIPG patients only) or definitive surgery, whichever is later. It is strongly recommended patients begin RT within 31 days of diagnosis/definitive surgery.
- If a biopsy was performed, the date of surgical biopsy will be considered the date of definitive diagnostic surgery; if a patient underwent two upfront surgeries \[e.g., biopsy then debulking\], this is the date of the second surgery)
- Patients must have received RT, but no other anti-cancer
Where
- Aurora, Colorado
- Washington D.C., District of Columbia
- Miami, Florida
- Atlanta, Georgia
- Ann Arbor, Michigan
- St Louis, Missouri
- Durham, North Carolina
- Cincinnati, Ohio
- Columbus, Ohio
- Philidelphia, Pennsylvania
- Houston, Texas
- Seattle, Washington
Collaborators
Accendatech USA Inc.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced May 15, 2026 · Source of record for eligibility and locations