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Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

15 Participants Needed

Olutasidenib for Chronic Myelomonocytic Leukemia

Overseen ByKelly Chien, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: M.D. Anderson Cancer Center

Disqualifiers: Hepatitis B, Hepatitis C, HIV, others

No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Prior Safety DataThis treatment has passed at least one previous human trial

Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

What is the purpose of this trial?

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

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications. However, hydroxyurea is allowed without a washout period, so you may be able to continue some medications. It's best to discuss your specific medications with the trial team.

How is the drug Olutasidenib unique for treating chronic myelomonocytic leukemia?

Olutasidenib is unique because it targets specific genetic mutations often found in chronic myelomonocytic leukemia, such as those affecting the epigenome and cell signaling pathways, which are not directly addressed by standard treatments like hypomethylating agents or stem cell transplantation.¹ ² ³ ⁴ ⁵

Research Team

Kelly Chien, MD

Principal Investigator

M.D. Anderson Cancer Center

Eligibility Criteria

This trial is for patients with specific blood disorders: Clonal Cytopenia of Undetermined Significance (CCUS), lower-risk Myelodysplastic Syndromes (MDS), or Chronic Myelomonocytic Leukemia (CMML). Participants must have a mutation in the IDH1 gene. Detailed eligibility criteria are not provided, but typically include age, health status, and previous treatments.

Inclusion Criteria

My cancer has a specific IDH1 mutation.

I am 18 years old or older.

My kidney function is within the required range.

See 7 more

Exclusion Criteria

I do not have any severe uncontrolled health or mental conditions.

Participant with white blood cell count > 25 x109/L

I cannot take pills by mouth or have a gut condition affecting drug absorption.

See 3 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive olutasidenib monotherapy, taking capsules twice daily

12 months

Regular visits as per study protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 weeks

Treatment Details

Interventions

Olutasidenib

Trial OverviewThe study is testing Olutasidenib's effectiveness in controlling CCUS, MDS, and CMML in patients with an IDH1 mutation. It's a Phase 2 trial which means it focuses on the drug’s efficacy and monitoring its safety.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: OlutasidenibExperimental Treatment1 Intervention

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.

Olutasidenib is already approved in United States for the following indications:

Approved in United States as Rezlidhia for:

Acute Myeloid Leukemia (AML) with a susceptible IDH1 mutation

Find a Clinic Near You

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+

Rigel Pharmaceuticals

Industry Sponsor

Trials

Recruited

4,000+

Findings from Research

Chronic myelomonocytic leukemia (CMML) has a poor prognosis with a median overall survival of about 2 years and a significant risk of transforming into acute myeloid leukemia (15-20% over 5 years), highlighting the need for better understanding and treatment options.

Current treatment options are limited, with only two FDA-approved drugs (5-azacitidine and decitabine) and allogeneic hematopoietic stem cell transplantation being the only curative approach, which carries high risks of complications.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Advances in chronic myelomonocytic leukemia and future prospects: Lessons learned from precision genomics.Mangaonkar, AA., Patnaik, MM.[2021]

Chronic myelomonocytic leukemia (CMML) is now recognized as a distinct category that combines features of myelodysplastic syndromes and myeloproliferative disorders, with a new prognostic score developed to classify patients into four groups based on their prognosis.

Recent research highlights the role of angiogenesis and specific genetic factors, such as tyrosine kinase fusion genes, in CMML, suggesting that targeted therapies like imatinib mesylate and antiangiogenic agents could be effective, although more studies are needed to explore these treatment options.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

CMML: a biologically distinct myeloproliferative disease.Cortes, J.[2006]

Chronic myelomonocytic leukemia (CMML) is characterized by specific gene mutations and cytogenetic abnormalities that can help stratify patients into risk groups, guiding treatment decisions; common mutations include TET2, SRSF2, and ASXL1, with ASXL1 mutations linked to worse outcomes.

Allogeneic stem cell transplantation is the preferred treatment for younger patients with poor prognostic factors, while hypomethylating agents may be considered for those ineligible for transplantation, highlighting the need for a consensus on treatment guidelines and response criteria for clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Chronic myelomonocytic leukemia prognostic classification and management: evidence base and current practice.Selimoglu-Buet, D., Solary, E.[2021]