ACT001 for Brain Cancer
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test the safety and effectiveness of ACT001, a new potential drug for treating brain cancers, specifically DIPG and H3K27-altered HGG. Participants will be divided into two groups: one with newly diagnosed DIPG patients and another with patients whose DIPG or H3K27-altered HGG has returned or worsened after initial treatment. This trial suits those who have completed radiation therapy for their brain cancer and are not currently on other anti-cancer medications. As a Phase 2 trial, the research measures how well the treatment works in an initial, smaller group, offering participants a chance to contribute to important advancements in brain cancer treatment.
Do I have to stop taking my current medications for the trial?
The trial requires that you stop taking any other anti-cancer agents and investigational drugs. If you are on corticosteroids, you may continue them if the dose is stable or decreasing. Some medications, like certain anticonvulsants and high-dose biotin, are not allowed, and SSRIs should be used with caution. Please discuss your specific medications with the study team to ensure eligibility.
Is there any evidence suggesting that ACT001 is likely to be safe for humans?
Research shows that ACT001 is under investigation for safety in treating brain tumors, such as DIPG and H3K27-altered HGG. Early results suggest that ACT001 can cross the blood-brain barrier, effectively reaching the brain. It blocks pathways that aid tumor growth, such as NF-κB and STAT3.
In earlier studies, ACT001 showed promising effects against tumors. However, as this is an ongoing Phase 2 study, researchers are still collecting complete safety data. Reaching this phase indicates that it has passed initial safety tests in humans, but further testing is needed to fully understand its safety.12345Why do researchers think this study treatment might be promising for brain cancer?
Unlike the standard treatments for brain cancer, such as radiation and chemotherapy, ACT001 offers a novel approach by targeting the underlying mechanisms of the disease. Researchers are excited about ACT001 because it specifically targets DIPG and H3K27-altered HGG tumors, which are notoriously difficult to treat with current options. Most treatments focus on destroying cancer cells broadly, but ACT001 works by interfering with specific pathways associated with tumor growth, potentially offering a more effective and less harmful alternative. This targeted mechanism may lead to better outcomes and fewer side effects, making it a promising option for patients with these challenging brain tumors.
What evidence suggests that ACT001 might be an effective treatment for brain cancer?
Research has shown that ACT001 may help treat brain cancer, particularly based on animal studies. In these studies, ACT001 increased the survival time of mice with tumors by 33%. This trial will test ACT001 in two separate groups: Cohort A, which includes patients with newly-diagnosed diffuse intrinsic pontine gliomas (DIPG) with typical MRI findings, and Cohort B, which includes patients with progressive, refractory, or recurrent DIPG or extra-pontine high-grade gliomas (HGG) with an H3K27 alteration. While these early results are promising, more studies are needed to determine its effectiveness in humans.23456
Who Is on the Research Team?
Sara Khan, MD, PhD, FRACP
Principal Investigator
Nationwide Children's Hospital
David S. Ziegler, MD, FRACP
Principal Investigator
Sydney Children's Hospitals Network
Peter de Blank, MD, MSCE
Principal Investigator
Children's Hospital Medical Center, Cincinnati
Are You a Good Fit for This Trial?
This trial is for patients with specific brain tumors: Diffuse Intrinsic Pontine Gliomas (DIPG) and H3K27-altered High Grade Gliomas (HGG). Detailed inclusion and exclusion criteria are not provided, but typically these would involve age, health status, prior treatments, and tumor characteristics.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive ACT001 at 875 mg/m2 orally BID for 28 days per cycle, up to 26 cycles
Follow-up
Participants are monitored for safety and effectiveness after treatment
Extension
Continuation of treatment beyond 26 cycles may be considered if patients are receiving clinical benefit
What Are the Treatments Tested in This Trial?
Interventions
- ACT001
Find a Clinic Near You
Who Is Running the Clinical Trial?
Nationwide Children's Hospital
Lead Sponsor
Accendatech USA Inc.
Industry Sponsor