Phase 2 Study of Olutasidenib for Patients with IDH1-mutated Blood Disorders

Sponsor: M.D. Anderson Cancer Center

Sponsor score: 0

ClinicalTrials ID:NCT06566742

Provider View

This study is testing if the drug olutasidenib can help manage certain blood disorders with an IDH1 mutation, such as CCUS, MDS, and CMML. The study will also look at the safety of the drug.

Patient Parameters

Program Overview

The study aims to find out if olutasidenib can help control blood disorders like Clonal Cytopenia of Undetermined Significance (CCUS), Myelodysplastic Syndromes (MDS), and Chronic Myelomonocytic Leukemia (CMML) that have a specific IDH1 mutation. Researchers will also study how safe the drug is for patients.

Description

The main goal of this study is to see how well olutasidenib works on its own in treating patients with certain blood disorders that have an IDH1 mutation. Researchers will also look at whether patients can avoid needing blood transfusions for at least 8 weeks, how safe the drug is, and if it helps patients live longer or prevents their condition from turning into leukemia. Additionally, the study will explore if the size of the IDH1 mutation in the blood decreases.

Researchers will also investigate other factors that might predict how well the treatment works or why it might not work, by looking at gene expression and other markers.

Eligibility Criteria

Inclusion Criteria

  • Have a confirmed diagnosis of CCUS or lower-risk MDS/CMML.
    • CCUS is defined by low blood cell counts for at least 30 days without other explanations and no signs of myeloid cancer.
    • Lower-risk MDS/CMML includes patients with specific low-risk scores.
  • Must have an IDH1 mutation with a variant allele frequency (VAF) of 0.02 or higher.
  • Be 18 years or older.
  • Have a performance status score of 0 to 2, meaning fully active or restricted in physically strenuous activity but ambulatory.
  • Have acceptable liver function, with specific limits on bilirubin and liver enzymes.
  • Have acceptable kidney function, with specific limits on creatinine levels.
  • Women of childbearing potential must have a negative pregnancy test.
  • Agree to use effective birth control during the study and for 90 days after the last dose.
  • Men must agree not to donate sperm, and women must agree not to donate eggs during the study and for 90 days after.
  • Be able and willing to sign informed consent before starting the study.

Exclusion Criteria

  • Cannot swallow pills or have conditions that affect absorption in the gut.
  • Have any uncontrolled serious medical condition, severe infection, or psychiatric illness that could increase risk.
  • Have active hepatitis B, hepatitis C, or HIV infection.
  • Pregnant or nursing women, or those not using effective birth control; men not using effective birth control.
  • Have a white blood cell count over 25 x 10^9/L (use of hydroxyurea is allowed to meet this criterion).
  • Unwilling or unable to follow study procedures or standard care.

Locations

FACILITYZIPCITYSTATE
MD Anderson Cancer Center77030HoustonTexas

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