ONC201 for Leukemia
What You Need to Know Before You Apply
What is the purpose of this trial?
This phase I/II trial studies the side effects and best dose of ONC201 and to see how well it works in treating patients with acute leukemia or high-risk myelodysplastic syndrome that has returned after a period of improvement (relapsed) or does not respond to treatment (refractory). ONC201 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
Will I have to stop taking my current medications?
The trial requires that you stop taking any standard or investigational treatments for your blood cancer at least 2 weeks before starting the study drug. If you're taking hydroxyurea, you must stop it at least 24 hours before starting the trial. The protocol does not specify other medications, but you should discuss your current medications with the trial team.
What makes the drug ONC201 unique for treating leukemia?
ONC201 is unique because it targets a specific pathway in cancer cells, potentially offering a new approach for treating leukemia compared to traditional therapies. While other treatments like histone deacetylase inhibitors focus on altering gene expression, ONC201 works through a different mechanism, which may provide benefits in cases where other treatments are less effective.12345
Who Is on the Research Team?
Gautam Borthakur, M.D.
Principal Investigator
M.D. Anderson Cancer Center
Are You a Good Fit for This Trial?
Adults over 18 with acute leukemia or high-risk myelodysplastic syndrome that's relapsed or refractory. They must be in fair health (ECOG 0-2), able to consent, and have no severe persistent side effects from past treatments. No recent heart issues, other cancer treatments within 3 years (except certain low-risk cases), or active infections like HIV/Hepatitis B/C.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive Akt/ERK inhibitor ONC201 orally in various dosing schedules depending on the assigned arm. Cycles repeat every 21 or 28 days in the absence of disease progression or unacceptable toxicity.
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- ONC201
Find a Clinic Near You
Who Is Running the Clinical Trial?
M.D. Anderson Cancer Center
Lead Sponsor
Oncoceutics, Inc.
Industry Sponsor