Olutasidenib + Hypomethylating Agents for Blood Disorders
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to determine if combining the drug olutasidenib with a hypomethylating agent (HMA) can help manage certain blood disorders, specifically MDS, CMML, and MPN. Researchers are also assessing the safety of this combination for patients. Suitable candidates for this trial have a confirmed IDH1 mutation and are experiencing higher-risk MDS, CMML, or advanced MPN that impacts their daily life. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group of participants.
Will I have to stop taking my current medications?
The trial requires that patients stop taking certain medications before starting the study. Specifically, patients must stop any prior chemotherapy at least one week before the study and must not be on JAK inhibitors at the start of the study. If you are taking other medications, the protocol does not specify, so it's best to discuss with the study team.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
In earlier studies, the combination of olutasidenib and azacitidine showed promising results for treating certain blood disorders. Research indicates that patients generally tolerate this combination well. Specifically, it led to high response rates and long-lasting remissions in patients with acute myeloid leukemia (AML), while keeping side effects manageable. Another study found that the safety of this combination matched expectations for each drug used individually.
These findings suggest that the combination is generally safe for people with blood disorders. However, like any treatment, side effects can occur. Participants should discuss potential risks and benefits with their healthcare provider before joining a trial.12345Why do researchers think this study treatment might be promising for blood disorders?
Researchers are excited about olutasidenib for blood disorders because it targets specific mutations in cancer cells, which can lead to more effective treatment outcomes. Unlike current hypomethylating agents like azacitidine or decitabine, which broadly target cell division, olutasidenib specifically inhibits mutant IDH1 enzymes that are often present in certain blood cancers. This targeted approach not only holds the promise of being more effective but also potentially reduces side effects by sparing healthy cells. Combining olutasidenib with azacitidine may enhance the treatment's overall efficacy, offering new hope for patients with blood disorders.
What evidence suggests that this treatment might be an effective treatment for blood disorders?
This trial will evaluate the combination of olutasidenib and azacitidine for treating certain blood disorders, such as MDS (myelodysplastic syndromes), CMML (chronic myelomonocytic leukemia), and MPN (myeloproliferative neoplasms). Studies have shown that using these drugs together is promising. Specifically, patients with an IDH1 mutation in MDS responded better and experienced longer disease-free periods with this combination. Research indicates that patients with higher-risk MDS and the IDH1 mutation lived for an average of 27.5 months when treated with both drugs, nearly doubling the survival time compared to those treated with azacitidine alone. Additionally, many patients achieved complete remission and no longer required blood transfusions. These findings suggest that this drug combination could effectively manage these challenging blood disorders.14678
Who Is on the Research Team?
Kelly Chien, MD
Principal Investigator
M.D. Anderson Cancer Center
Are You a Good Fit for This Trial?
This trial is for patients with certain blood disorders like higher-risk myelodysplastic syndromes, chronic myelomonocytic leukemia, or advanced myeloproliferative neoplasm that have a specific mutation called IDH1. Detailed eligibility criteria are not provided.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive olutasidenib in combination with a hypomethylating agent. Olutasidenib is taken twice daily, and Azacitidine is administered IV or SubQ for 7 days every 28 days.
Follow-up
Participants are monitored for safety and effectiveness after treatment, including overall survival and progression-free survival.
What Are the Treatments Tested in This Trial?
Interventions
- Azacitidine
- Decitabine
- Olutasidenib
Olutasidenib is already approved in United States for the following indications:
- 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
Rigel Pharmaceuticals
Industry Sponsor