A Phase 2 Study Evaluating Olutasidenib in Patients With IDH1-mutated Clonal Cytopenia of Undetermined Significance and Lower-risk Myelodysplastic/Syndromes/Chronic Myelomonocytic Leukemia.

The film gives the main points. The full list below is copied from the registry; the study team makes the final decision.

• Age18 Years to no maximum
• SexAll
• Healthy volunteersAccepted

You may be able to join if

• To be considered eligible to participate in this study, a patient must meet ALL inclusion criteria as follows:
• Pathologically proven CCUS or lower-risk MDS/CMML.
• CCUS is defined as the presence of cytopenia (absolute neutrophil count < 1.8 x 10^9/L, hemoglobin < 13 g/dL in males or < 12 g/dL in females, and/or platelets < 150 x 10^9/L) for at least 30 days that are otherwise unexplained and with no diagnostic hematopathologic features of myeloid neoplasms. Patients with known Duffy-null phenotype must have absolute neutrophil counts less than their lower limit of normal.
• Lower-risk MDS/CMML includes patients with International Prognostic Scoring System (IPSS) low- or intermediate-1-risk disease and Revised IPSS (IPSS-R) score ≤ 3.5 and Molecular IPSS (IPSS-M) very low-, low-, or moderate low-risk categories.
• Patients must have a documented IDH1 mutation with variant allele frequency (VAF) ≥ 0.02.
• Patients ≥ 18 years old.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 (Appendix A)
• Acceptable liver function
• Bilirubin ≤ 2 times upper limit of normal (ULN) or ≤ 3 times ULN in patients with Gilbert Syndrome.
• Aspartate transaminase (AST), alanine transaminase (ALT), and alkaline phosphatase ≤ 3 times ULN.
• Acceptable renal function with serum creatinine ≤ 1.5 times ULN or calculated creatinine clearance ≥ 50 mL/min (as assessed by Cockcroft-Gault, Modification of Diet in Renal Disease Formula [MDRD], or Chronic Kidney Disease Epidemiology [CKD-Epi] validated measures).
• Negative serum or urine pregnancy test if female of childbearing potential.
• For fertile men and women, agreement to use highly effective contraceptive methods for the duration of study participation and 90 days after the last dose of study medication. Appropriate highly effective method(s) of contraception include oral or injectable hormonal birth control, intrauterine device (IUD), and double barrier methods (for example a condom in combination with a spermicide).
• Agreement for male patients not to donate sperm and for female patients of childbearing potential not to donate ova during the study and for 90 days after the final dose of study drug.
• Ability and willingness to signed informed consent prior to beginning study and undergoing procedures.

You cannot join if

• To be eligible for entry into the study, the patient must NOT meet any of the exclusion criteria listed below:
• Patients unable to swallow oral medications, or patients with gastrointestinal conditions (e.g., malabsorption, resection, etc.) deemed by the Investigator to jeopardize intestinal absorption.
• Patients with any concurrent uncontrolled clinically significant medical condition, including life-threatening severe infection or psychiatric illness, which could place the patient at unacceptable risk of study treatment.
• Known active hepatitis B (hepatitis B virus [HBV]) or hepatitis C (hepatitis C virus [HCV]) or HIV infection.
• Pregnant or nursing women or women of childbearing potential not using highly effective contraception; male patients not using highly effective contraception as defined in the inclusion criteria.
• Subject with white blood cell count > 25 x10^9/L.
• Note: hydroxyurea use is permitted to meet this criterion with no washout required.
• Unwillingness or inability to comply with procedures either required in this protocol or considered standard of care.

This is how the treatment is meant to work. Whether it helps, and how safe it is, is what this trial is testing.

olutasidenib. An orally available inhibitor of isocitrate dehydrogenase type 1 (IDH1; IDH-1; IDH1 [NADP+] soluble) with a mutation at arginine (R) 132, IDH1(R132), with potential antineoplastic activity. Upon administration, olutasidenib specifically inhibits IDH1(R132), thereby inhibiting the formation of the oncometabolite 2-hydroxyglutarate (2HG) from alpha-ketoglutarate (a-KG). This prevents 2HG-mediated signaling and leads to both an induction of cellular differentiation and an inhibition of cellular proliferation in tumor cells expressing IDH(R132). IDH1(R132) mutations are highly expressed in certain malignancies, including gliomas; they initiate and drive cancer growth by both blocking cell differentiation and catalyzing the formation of 2HG. NCI Drug Dictionary

Treatments listed in the registry

• Olutasidenib (Drug): Given by PO

Na · Single Group · masking: None

As the registry describes them. Requirements to join, such as a tissue sample, are in the eligibility list above even when on-study procedures are optional. Ask the study team which steps apply to you.

• biopsy: Part of the study plan
• bone marrow: Part of the study plan
• blood samples: Part of the study plan

Main goals

• Safety and adverse events (AEs) (Through study completion; an average of 1 year.)

Planned enrollment: 15 (estimated).

• University of Miami, Miami, Florida
• MD Anderson Cancer Center, Houston, Texas

Site status as of Oct 30, 2026. Check the live record before contacting a site.

• ClinicalTrials.gov NCT06566742
• NCI Drug Dictionary: C129687

Registry record reviewed as fetched Oct 30, 2026, last updated by the sponsor Sep 10, 2026. Public record; not medical advice.