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

NCT06566742 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 2024-0509NCI-2024-07045
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

To learn if olutasidenib can help to control CCUS, MDS, and/or CMML. The safety of the drug will also be studied.

Conditions

Myelodysplastic Syndromes; Chronic Myelomonocytic Leukemia; Clonal Cytopenia of Undetermined Significance

Outcome Measures

Primary Outcomes (1)

• Safety and adverse events (AEs)

  Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0

  Through study completion; an average of 1 year.

Study Arms (1)

Olutasidenib

EXPERIMENTAL

Participants will take capsules of olutasidenib 2 times each day while you are on study. Each dose should be taken about 12 hours apart at least 1 hour before or 2 hours after a meal.

Drug: Olutasidenib

Interventions

Olutasidenib DRUG

Given by PO

Olutasidenib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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 may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• Rigel Pharmaceuticals: collaborator

Study Sites (1)

MD Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Related Links

• MD Anderson Cancer Center

MeSH Terms

Conditions

Myelodysplastic Syndromes; Leukemia, Myelomonocytic, Chronic

Interventions

olutasidenib

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myelodysplastic-Myeloproliferative Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Kelly Chien, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Kelly Chien, MD

CONTACT

(713) 745-7584; kchien@mdanderson.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 20, 2024
• First Posted: August 22, 2024
• Study Start: December 10, 2024
• Primary Completion (Estimated): August 31, 2027
• Study Completion (Estimated): August 31, 2029
• Last Updated: March 4, 2026

Record last verified: 2026-03

Locations

US (1)