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A Phase 2 Study Evaluating Olutasidenib in Combination With Hypomethylating Agents in Patients With IDH1-mutated Higher-risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Advanced Myeloproliferative Neoplasm | Clinical Trials | Clareo Health

RECRUITINGPHASE2

A Phase 2 Study Evaluating Olutasidenib in Combination With Hypomethylating Agents in Patients With IDH1-mutated Higher-risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Advanced Myeloproliferative Neoplasm

NCT06597734•M.D. Anderson Cancer Center

View on ClinicalTrials.gov

Summary

To learn if olutasidenib, when combined with a drug called a hypomethylating agent (HMA) can help to control MDS, CMML, and/or MPN. The safety of the drug combination will also be studied.

Detailed Description

Primary Objectives To determine the overall response rate of olutasidenib in combination with investigator's choice of HMA in patients with IDH1-mutated higher-risk MDS/CMML or advanced MPN Secondary Objectives The secondary objectives of this study are: * To evaluate the rates of complete remission (CR) and median duration of CR * To ascertain the safety and tolerability of olutasidenib with HMA in this participant population * To determine survival including overall survival (OS), progression-free survival (PFS), and duration of response (DOR) To analyze reduction in IDH1 clone size Exploratory Objectives * To examine overall response rate of patients previously exposed to venetoclax * To investigate global gene expression profiles, DNA methylation profiles, and other potential prognostic markers to explore predictors of antitumor activity and/or resistance to treatment

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Pathologically proven higher-risk MDS/CMML or advanced MPN
•1. MDS participants must have International Prognostic Scoring System (IPSS) intermediate-2- or high-risk disease or Revised IPSS (IPSS-R) score \>= 3.5 or Molecular IPSS (IPSS-M) moderate high-, high-, or very high-risk disease or bone marrow blast percentage.
•2. CMML patients must have CPSS-Mol int-1, int-2, or high-ris disease (Elena C et al, Blood 2016)
•3. Advanced MPN is defined as bone marrow blast percentage \>=/=10%.
•4. Participants on the treatment-naive arm must not have received a prior HMA. Agents such as growth factors (e.g. erythropoietin stimulating agents, luspatercept, eltrombopag, granulocyte colony stimulating factors), cyclosporine, and/or hydroxyurea are allowed.
•5. Patients on the previously-treated arm must have received a prior HMA and/or ivosidenib. Prior stem cell transplantation in allowed\>
•2. Participants must have a documented IDH1 mutation
•3. Patients with previously-treated MDS must be ineligible to receive treatment with ivosidenib or have progressed on treatment with ivosidenib.
•4. Participants \>=/= 18 years old
•5. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 (Appendix A)
+6 more criteria

Exclusion Criteria

•1. Participants unable to swallow oral medications, or participants with gastrointestinal conditions (e.g., malabsorption, resection, etc.) deemed by the Investigator to jeopardize intestinal absorption
•2. Participants with any concurrent uncontrolled clinically significant medical condition, including life-threatening severe infection or psychiatric illness, which could place the participant at unacceptable risk of study treatment
•3. Patients must have discontinued prior chemotherapy at least 1 week prior to the start of study treatment. Patients with MPN must be off JAK inhibitors at the start of study treatment
•4. Patients with active graft-versus-host-disease (GVHD) status post stem cell transplantation, including active chronic GVHD requiring topical therapy. Patients must have discontinued calcineurin inhibitors at least 4 weeks prior to the start of study treatment
•5. Known active hepatitis B (HBV) or hepatitis C (HCV) or HIV infection
•6. Pregnant or nursing women or women of childbearing potential not using highly effective contraception; male participants not using highly effective contraception
•7. Participants with white blood cell count \> 25 x109/L Note: hydroxyurea use is permitted to meet this criterion
•8. Unwillingness or inability to comply with procedures either required in this protocol or considered standard of care

Locations (1)

MD Anderson Cancer Center

Houston, Texas, United States

Key Dates

Start Date

January 28, 2025

Primary Completion Date

August 31, 2027

Completion Date

August 31, 2029

Last Updated

October 9, 2025

Enrollment

ESTIMATED participants

Conditions

Chronic Myelomonocytic LeukemiaAdvanced Myeloproliferative NeoplasmsIDH1-mutated Higher-Risk Myelodysplastic Syndromes

Interventions

Olutasidenib

DRUG

Contacts

Kelly Chien, MD

CONTACT

713-745-7584 Kchien@mdanderson.org

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 9, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

A Phase 2 Study Evaluating Olutasidenib in Combination With Hypomethylating Agents in Patients With IDH1-mutated Higher-risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Advanced Myeloproliferative Neoplasm

Inclusion Criteria

Exclusion Criteria

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