RecruitingPhase 2

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 join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 18 months, 3 years
  • Procedures mentioned: Blood draws, Biopsies

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Patients with CCUS receive up to 18 months of olutasidenib."
  • "Additionally, patients undergo blood sample collection and bone marrow aspiration and biopsy on study."
  • "After completion of study treatment, patients are followed up every 3 months for up to 3 years."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests whether olutasidenib can help control certain blood conditions: CCUS, lower-risk MDS, or lower-risk CMML. It is for adults whose blood cells have a change in the IDH1 gene. The study will also check the drug’s safety.

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

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be age 18 or older.
  • Have confirmed CCUS (unexplained low blood cell counts for at least 30 days), or MDS or CMML (blood cancers) that meet the study’s lower-risk scores.
  • Have a confirmed IDH1 gene change at a level of at least 2%.
  • Be able to do self-care and be out of bed or a chair for at least half of waking hours.
  • Have liver and kidney test results within the study’s limits.
  • Have a negative pregnancy test if you can become pregnant.
  • If able to have children, agree to use highly effective birth control and not donate sperm or eggs during the study and for 90 days after the last dose.
  • Be able and willing to sign a consent form before starting study tests or treatment.

Who can't join

  • Cannot swallow pills, or have a gut condition that could keep your body from taking in the drug.
  • Have an uncontrolled serious health condition, such as a severe infection or mental illness, that could make treatment unsafe.
  • Have known active hepatitis B or C (liver infections), or HIV.
  • Are pregnant, breastfeeding, or not using the required birth control.
  • Have a white blood cell count above 25 billion per liter. Hydroxyurea may be used to lower the count to meet this limit.
  • Are unable or unwilling to follow study procedures or usual care procedures.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: To be considered eligible to participate in this study, a patient must meet ALL inclusion criteria as follows: 1. Pathologically proven CCUS or lower-risk MDS/CMML. 1. 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. 2. 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. 2. Patients must have a documented IDH1 mutation with variant allele frequency (VAF) ≥ 0.02. 3. Patients ≥ 18 years old. 4. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 (Appendix A) 5. Acceptable liver function 1. Bilirubin ≤ 2 times upper limit of normal (ULN) or ≤ 3 times ULN in patients with Gilbert Syndrome. 2. Aspartate transaminase (AST), alanine transaminase (ALT), and alkaline phosphatase ≤ 3 times ULN. 6. 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). 7. Negative serum or urine pregnancy test if female of childbearing potential. 8. 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). 9. 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. 10. Ability and willingness to signed informed consent prior to beginning study and undergoing procedures. Exclusion Criteria: To be eligible for entry into the study, the patient must NOT meet any of the exclusion criteria listed below: 1. Patients unable to swallow oral medications, or patients with gastrointestinal conditions (e.g., malabsorption, resection, etc.) deemed by the Investigator to jeopardize intestinal absorption. 2. 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. 3. Known active hepatitis B (hepatitis B virus \[HBV\]) or hepatitis C (hepatitis C virus \[HCV\]) or HIV infection. 4. 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. 5. Subject with white blood cell count \> 25 x10\^9/L. * Note: hydroxyurea use is permitted to meet this criterion with no washout required. 6. Unwillingness or inability to comply with procedures either required in this protocol or considered standard of care.

Study sites (2)

FL

TX