Actively Recruiting
ONC201 in Treating Patients With Relapsed or Refractory Acute Leukemia or High-Risk Myelodysplastic Syndrome
Led by M.D. Anderson Cancer Center · Updated on 2026-04-14
120
Participants Needed
1
Research Sites
577 weeks
Total Duration
On this page
Sponsors
M
M.D. Anderson Cancer Center
Lead Sponsor
O
Oncoceutics, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
ONC201 in Treating Patients With Relapsed or Refractory Acute Leukemia or High-Risk Myelodysplastic Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must have relapsed or refractory acute leukemias or high-risk myelodysplastic syndrome without expected durable remission from standard therapies
- Age 18 years or older
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2
- Women who can become pregnant must use effective contraception for 16 weeks after last study dose and have a negative pregnancy test within 1 week before treatment; nursing women are excluded
- Sexually active men must use effective contraception during and for 16 weeks after study treatment
- Able and willing to give written informed consent
- At least 2 weeks since prior cytotoxic treatment or at least 5 half-lives since noncytotoxic treatment; must be off hydroxyurea for at least 24 hours before starting study treatment
- Persistent toxicities from prior therapy must be grade 1 or less
- Serum creatinine less than 2.0 mg/dl
- Total bilirubin less than or equal to 1.5 times the upper limit of normal unless due to Gilbert's syndrome
- ALT or AST less than or equal to 3 times the upper limit of normal unless due to organ leukemic involvement
- More than 6 months since autologous or allogeneic stem cell transplant with no active graft-versus-host disease above grade 1 and no high dose steroid or immunosuppressive therapy except low dose cyclosporine or tacrolimus
You will not qualify if you...
- Uncontrolled illness such as infection, severe heart failure, uncontrolled arrhythmia, or psychiatric illness limiting study compliance
- Active heart disease including recent myocardial infarction within 3 months, symptomatic coronary artery disease, uncontrolled arrhythmias, or severe congestive heart failure
- Received any other standard or investigational treatment for blood cancer within past 2 weeks or 5 half-lives for noncytotoxic agents
- Diagnosed or treated for another cancer within past 3 years except certain low-risk cancers after curative therapy
- Known HIV, hepatitis C infection, or hepatitis B carrier status
- Active drug use or alcoholism
- Known or active central nervous system involvement of leukemia
- White blood cell count above 25 x 10^9/L before starting venetoclax
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
M D Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
5
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