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ONC201 for Relapsed/Refractory Acute Leukemia or High-Risk MDS

This study is testing ONC201, alone or with Venetoclax, for adults (18+) with acute leukemia or high-risk myelodysplastic syndrome (MDS) that has come back or isn't responding to treatment. ONC201 may work by blocking enzymes (proteins that speed up chemical reactions) cancer cells need to grow. Researchers want to find the best dose of ONC201, understand its side effects, and see how well it shrinks cancer. Success would mean finding a safe dose and seeing the cancer respond to the treatment. The study is currently recruiting about 120 participants, but its overall status is unclear.

Study design
This is a Phase I/II interventional study with an estimated enrollment of 120 participants. It is a dose-escalation study followed by a Phase II study, with patients assigned to one of five arms.
What's involved
Patients receive ONC201 orally once every three weeks. The study aims to measure the maximum tolerated dose within 21 days and objective response within 63 days.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoints are measured at 21 days for maximum tolerated dose and 63 days for objective response. Longer-term follow-up is not specified.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT02392572

ONC201 in Treating Patients With Relapsed or Refractory Acute Leukemia or High-Risk Myelodysplastic Syndrome

Terminated
PHASE1Ages 18+InterventionalTreatment
M.D. Anderson Cancer Center
~58 participants
Updated 2026-08-20 on ClinicalTrials.gov
What's tested:Akt/ERK Inhibitor ONC201Venetoclax

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Maximum Tolerated Dose (MTD) of ONOC201 in Relapsed or Refractory Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS) or Acute Lymphoblastic Leukemia (ALL)
Measured over 21 days
+1 more outcome measured

Conditions

Recurrent Acute Lymphoblastic Leukemia
Recurrent Acute Myeloid Leukemia
Recurrent Myelodysplastic Syndrome
Refractory Acute Lymphoblastic Leukemia
Refractory Acute Myeloid Leukemia
Refractory Myelodysplastic Syndrome

Explore related trials

Acute Lymphoblastic LeukemiaAcute Myeloid LeukemiaMyelodysplastic Syndrome

Where it's being run

1 sites across 1 states
Texas1

Study leadership

  • Gautam Borthakur · PRINCIPAL_INVESTIGATOR · M.D. Anderson Cancer Center

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

For Arms A, B, C, D, E, F patients must have relapsed or refractory acute leukemias or high-risk MDS for which no standard therapies are anticipated to result in a durable remission
Age \>= 18 years
Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) must use acceptable contraceptive methods (abstinence, intrauterine device \[IUD\], oral contraceptive or double barrier device, such as a condom, diaphragm, or cervical/vault cap), for 16 weeks after the last dose of study drug, and must have a negative serum or urine pregnancy test within 1 week prior to beginning treatment on this trial; nursing patients are excluded; sexually active men must also use acceptable contraceptive methods for the duration of time on study and for at least 16 weeks after the last dose of study drug; pregnant and nursing patients are excluded because the effects of ONC201on a fetus or nursing child are unknown
Must be able and willing to give written informed consent
The interval from prior treatment to time of study drug administration should be at least 2 weeks for cytotoxic agents or at least 5 half-lives for noncytotoxic agents; if the patient is on hydroxyurea to control peripheral blood leukemic cell counts, the patient must be off hydroxyurea for at least 24 hours before initiation of treatment on this protocol; persistent clinically significant toxicities from prior therapy must not be greater than grade 1
Serum creatinine \< 2.0 mg/dl
Total bilirubin =\< 1.5 x the upper limit of normal (ULN) unless considered due to Gilbert's syndrome
Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) =\< 3 x the ULN unless considered due to organ leukemic involvement
Relapse \> 6 months since autologous or allogeneic stem cell transplantation provided:
No active graft-versus-host disease (GVHD \> grade 1)
No treatment with high dose steroids for GVHD (up to \>= 20 mg prednisolone or equivalent per day)
No treatment with immunosuppressive drugs with the exception of low dose cyclosporine and tacrolimus

Exclusion

Uncontrolled intercurrent illness including, but not limited to uncontrolled infection, symptomatic congestive heart failure (New York Heart Association class III and IV), uncontrolled cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
Active heart disease including myocardial infarction within previous 3 months, symptomatic coronary artery disease, arrhythmias not controlled by medication, or uncontrolled congestive heart failure (New York Heart Association class III and IV)
Patients receiving any other standard or investigational treatment for their hematologic malignancy within past 2 weeks for cytotoxic agents or at least 5 half-lives for noncytotoxic agents
Subject has been diagnosed or treated for another malignancy within 3 years of enrolment, except in situ malignancy, or low-risk prostate, skin or cervix cancer after curative therapy
Known history of seropositive for human immunodeficiency virus (HIV) antibodies (HIV1 and HIV2), hepatitis C antibody (Hep C Ab) or a hepatitis B carrier (positive for hepatitis B surface antigen \[HBsAg\])
Active drug use or alcoholism
Known or active central nervous system (CNS) involvement by leukemia
White blood cell count more than 25 x 109/L prior to initiation of venetoclax

What this trial measures

  • Maximum Tolerated Dose (MTD) of ONOC201 in Relapsed or Refractory Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS) or Acute Lymphoblastic Leukemia (ALL)21 days

    MTD is defined as the highest dose level in which 6 patients have been treated with at most 1 instance of dose limiting toxicity (DLT). Toxicities defined according to Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. DLT defined as a clinically significant adverse event or abnormal laboratory value assessed as unrelated to disease progression, intercurrent illness, or concomitant medications and occurring during the first cycle on study that meets any of the following criteria: CTCAE grade 3 AST (SGOT) or ALT (SGPT) for \> 7 days CTCAE grade 4 AST (SGOT) or ALT (SGPT) of any duration All other clinically significant NCI common terminology criteria that are CTCAE grade 3 or 4 (except for electrolyte disturbances responsive to correction within 24 h, diarrhea, nausea and vomiting that responds to standard medical care)

  • Objective Response (OR) (Phase II)63 days

    Objective responses for patients with AML and ALL include Complete Remission (CR), Complete Remission with Incomplete Blood Count Recovery (CRi), Partial Remission (PR) and morphologic leukemia-free state(Cheson and others, 2003).