Actively Recruiting
A Phase 2 Study Evaluating Olutasidenib in Patients With IDH1-mutated Clonal Cytopenia of Undetermined Significance and Lower-risk Myelodysplastic/Syndromes/Chronic Myelomonocytic Leukemia
Led by M.D. Anderson Cancer Center · Updated on 2026-03-04
15
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
Sponsors
M
M.D. Anderson Cancer Center
Lead Sponsor
R
Rigel Pharmaceuticals
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating olutasidenib in patients with IDH1-mutated Clonal Cytopenia of Undetermined Significance (CCUS), lower-risk Myelodysplastic Syndromes (MDS), and Chronic Myelomonocytic Leukemia (CMML). The study aims to determine how well olutasidenib controls these conditions, assess its safety, and investigate various outcomes including transfusion independence, survival, leukemia transformation rates, and changes in IDH1 clone size. The study also explores gene expression and DNA methylation to identify potential predictors of treatment response or resistance. Participants receive olutasidenib capsules taken orally twice daily, about 12 hours apart and separated from meals. Each treatment cycle lasts 28 days and continues without disease progression or unacceptable side effects. Patients with CCUS may receive treatment for up to 18 months, while those with lower-risk MDS or CMML can continue treatment until disease progression or intolerable toxicity. Blood samples and bone marrow biopsies are collected during the study to monitor disease and treatment effects. After finishing treatment, participants are followed every 3 months for up to 3 years to track long-term outcomes. Throughout the study, safety and adverse events are closely monitored. Researchers measure response rates, transfusion independence, changes in disease markers, and survival. This involvement includes regular blood tests, bone marrow evaluations, and ongoing assessments of health status.
CONDITIONS
Brief Title
A Phase 2 Study Evaluating Olutasidenib in Patients With IDH1-mutated Clonal Cytopenia of Undetermined Significance and Lower-risk Myelodysplastic/Syndromes/Chronic Myelomonocytic Leukemia.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Pathologically confirmed diagnosis of Clonal Cytopenia of Undetermined Significance (CCUS) or lower-risk Myelodysplastic Syndromes (MDS)/Chronic Myelomonocytic Leukemia (CMML)
- For CCUS: cytopenia for at least 30 days without other causes and no diagnostic myeloid neoplasm features
- For lower-risk MDS/CMML: International Prognostic Scoring System (IPSS) low or intermediate-1 risk, Revised IPSS score 3.5, or Molecular IPSS very low, low, or moderate low risk
- Documented IDH1 mutation with variant allele frequency 0.02
- Age 18 years or older
- Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
- Bilirubin 2 times upper limit of normal (ULN) or 3 times ULN if Gilbert Syndrome
- AST, ALT, and alkaline phosphatase 3 times ULN
- Serum creatinine 1.5 times ULN or creatinine clearance 50 mL/min
- Negative pregnancy test if female of childbearing potential
- Agreement to use highly effective contraception during study and 90 days after last dose
- Agreement not to donate sperm or ova during study and 90 days after last dose
- Ability and willingness to provide informed consent
You will not qualify if you...
- Unable to swallow oral medications or have gastrointestinal conditions affecting absorption
- Uncontrolled serious medical conditions including severe infections or psychiatric illnesses
- Active hepatitis B, hepatitis C, or HIV infection
- Pregnant or nursing women or women not using effective contraception; men not using effective contraception
- White blood cell count greater than 25 x 10^9/L (hydroxyurea use allowed to meet this criterion)
- Unwilling or unable to comply with study procedures or standard care
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
screening and enrollment visit
Duration - Up to 18 months for participants with CCUS; until disease progression or unacceptable toxicity for participants with lower-risk MDS/CMML
Participants take olutasidenib capsules orally twice daily in 28-day cycles until disease progression or unacceptable toxicity. Those with CCUS receive treatment for up to 18 months.
Repeated 28-day cycles
Duration - Up to 3 years
After completing treatment, participants are followed every 3 months for up to 3 years to monitor health status.
Visits every 3 months
Trial Site Locations
Total: 1 location
1
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
K
Kelly Chien, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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