What type of study is this?

This is a Human Clinical Trial study (also classified as: Phase 1, Pre-Registered Trial).

What question did this study set out to answer?

To assess the safety and efficacy of ONC201 as a post-transplant maintenance treatment for AML and MDS.

December 8, 2025BloodOpen Access

Phase I trial of a novel first-in-class drug ONC201 as a post-transplant maintenance for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)

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Authors

LMLori J. ManessNebraska Medical Center KGKrishna GundaboluUniversity of Nebraska Medical Center

Discussion

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Implication

Phase 1 trial shows ONC201 maintains safety and may improve survival in AML and MDS post-transplant patients, suggesting further development is warranted.

Key Points

To assess the safety and efficacy of ONC201 as a post-transplant maintenance treatment for AML and MDS.
Phase 1 trial with a 3+3 design
Patients received oral ONC201 weekly for up to 13 cycles
Primary objective to determine dose-limiting toxicities during treatment
No dose-limiting toxicities observed
2-year overall survival rate of 70%
Cytopenias occurred infrequently, with 15% anemia and 10% neutropenia

Cite This Study

Maness et al. (2025) studied this question.

synapsesocial.com/papers/69362f6c4fa91c937236dfc5 https://doi.org/10.1182/blood-2025-174

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Implication

Phase 1 trial shows ONC201 maintains safety and may improve survival in AML and MDS post-transplant patients, suggesting further development is warranted.

Phase I trial of a novel first-in-class drug ONC201 as a post-transplant maintenance for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)

Discussion

Member takes

Implication

Key Points

Cite This Study

Phase I trial of a novel first-in-class drug ONC201 as a post-transplant maintenance for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)

Discussion

Member takes

Implication

Key Points

Cite This Study

Also Consider

Also Consider