A new medicine called ONC201 for acute myeloid leukemia

Ph1/2 Study of the Imipridone ONC201 for Treatment of AML IND125,203 (12/23/2014)

['FUNDING_R01'] · UNIVERSITY OF TX MD ANDERSON CAN CTR · NIH-10891412

This research explores a new medicine, ONC201, to improve treatment for people with acute myeloid leukemia (AML).

Quick facts

Phase['FUNDING_R01']
Study typeNih_funding
SexAll
SponsorUNIVERSITY OF TX MD ANDERSON CAN CTR (nih funded)
Locations1 site (HOUSTON, UNITED STATES)
Trial IDNIH-10891412 on ClinicalTrials.gov

What this research studies

We are conducting a clinical trial to learn more about ONC201, a new type of medicine that works by targeting a specific survival mechanism within leukemia cells. Our earlier laboratory work showed that ONC201 can cause AML cells to die without harming healthy cells, and it appears especially effective against AML that is resistant to other treatments. We also found that ONC201 may eliminate leukemia stem cells, which are often responsible for the disease coming back. Early results from other trials suggest ONC201 is well-tolerated and shows promising signs of activity.

Who could benefit from this research

Good fit: Ideal candidates for this opportunity would be patients diagnosed with acute myeloid leukemia (AML).

Not a fit: Patients without acute myeloid leukemia would not receive direct benefit from this specific treatment.

Why it matters

Potential benefit: If successful, this medicine could lead to higher cure rates for acute myeloid leukemia, particularly for patients whose disease is resistant to current therapies.

How similar studies have performed: Early trials of ONC201 have shown good tolerability and initial signs of clinical response.

Where this research is happening

HOUSTON, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.