Study on ACT001 for Treating Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas
Sponsor: Nationwide Children's Hospital
ClinicalTrials ID:NCT06838676
This clinical trial is testing the safety and effectiveness of a drug called ACT001 in treating two types of brain tumors: Diffuse Intrinsic Pontine Gliomas (DIPG) and H3K27-altered High Grade Gliomas (HGG).
Patient Parameters
| Parameter | Options |
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Program Overview
This Phase II study aims to find out if ACT001 is safe and effective for patients with DIPG and H3K27-altered HGG. The study is open-label, meaning both the researchers and participants know what treatment is being given. Participants will be divided into two groups: those newly diagnosed with DIPG and those with progressive or recurrent DIPG or H3K27-altered HGG. They will receive ACT001 in cycles of 28 days, with the possibility of continuing treatment for up to two years if they benefit from it.
Description
Participants will be placed in one of two groups: Cohort A for newly diagnosed DIPG patients, and Cohort B for those with progressive or recurrent DIPG or H3K27-altered HGG. Each group will receive ACT001 orally twice a day for 28 days, which is considered one cycle of treatment. The dose will start at 875 mg/m2 and can go up to 1700 mg twice a day. If participants are benefiting from the treatment, they can continue for up to 26 cycles (about two years) or until the disease progresses. Treatment beyond 26 cycles may be considered if it continues to be beneficial, as decided by the sponsor and doctor.
Eligibility Criteria
Inclusion Criteria
- Age between 1 and 39 years.
- Diagnosed with either newly diagnosed DIPG or progressive/recurrent DIPG or H3K27-altered HGG.
- For newly diagnosed DIPG: Must have completed radiation therapy within 28 to 35 days before starting the study.
- For progressive/recurrent DIPG or H3K27-altered HGG: Must have measurable disease and completed prior radiation therapy.
- Must be in good general health with a performance score of 50% or higher.
- Must have adequate organ function, including bone marrow, kidney, liver, and heart function.
- Must have a life expectancy of at least 3 months.
- Must be able to provide informed consent and agree to follow study procedures.
Exclusion Criteria
- For Cohort A: Patients with metastatic disease are not eligible.
- Currently taking other anti-cancer treatments or investigational drugs.
- Have uncontrolled infections or have had a solid organ transplant.
- Pregnant or breastfeeding women are not eligible.
- Must agree to use effective birth control during the study and for 3 months after.
- Unable to comply with study requirements or have previously received ACT001 or parthenolide.
Locations
| FACILITY | ZIP | CITY | STATE |
|---|---|---|---|
| Children's National Medical Center | 20010 | Washington | District of Columbia |
| Nicklaus Children's Hospital | 33155 | Miami | Florida |
| Emory University/Children's Healthcare of Atlanta | 30322 | Atlanta | Georgia |
| C.S. Mott Children's Hospital | 48109 | Ann Arbor | Michigan |
| St. Louis Children's Hospital | 63110 | St. Louis | Missouri |
| Duke University Medical Center | 27708 | Durham | North Carolina |
| Cincinnati Children's Hospital | 45229 | Cincinnati | Ohio |
| Nationwide Children's Hospital | 43235 | Columbus | Ohio |
| Children's Hospital of Philidelphia | 19104 | Philidelphia | Pennsylvania |
| Texas Children's Hospital | 77030 | Houston | Texas |
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