Brief Summary
This study will enroll participants with myelodysplastic syndromes (MDS) with an Isocitrate dehydrogenase protein, 1 (IDH1) mutation, who have not received treatment with a hypomethylating agent previously. Participants will be randomized to receive either ivosidenib (IVO) alone or azacitidine (AZA) alone. IVO will be administered daily throughout the 28-day treatment cycle and AZA will be administered for the first 7 days of each 28-day cycle. Study visits will be conducted every week during Cycle 1 (Days 1, 8, 15, and 22), and Day 1 of each cycle thereafter. After the last dosethe amount of medication taken of treatment, participants will attend an safety follow-up visit and participants will be followed to assess overall survival. Study visits may include a bone marrowsoft, spongy tissue found in bones that makes blood cells aspirateto draw out fluid or tissue from the body, often with a syringe, physical exam, echocardiograma type of ultrasound that uses sound waves to create detailed images of the heart to assess heart structure, function and blood flow (ECHO), electrocardiogram (ECG), bloodthe red bodily fluid that transports oxygen and other nutrients around the body and urine analysis, and questionnaires.
Intervention / Treatment
- Drug: Ivosidenib
- Drug: Azacitidine
Inclusion Criteria:
- Diagnosisthe process of identifying a disease based on signs and symptoms, patient history and medical test results of HMA naive IDH1 R132 mutated MDS defined according to WHO criteria (5th edition):
- Moderate high, high and very high-risk MDS per IPSS-M score will be eligible regardless of blood counts and with blast counts 0-19%.
- Low and moderate low-risk MDS per IPSS-M score must:
- Have cytopenias related to MDS, defined as: <100 plateletssmall disc-shaped blood cells that clump together to form clots to stop bleeding/microliter, or absolute neutrophil count (ANC) <1000/mm3, or hemoglobin <10g/dL AND
- Have a blast count between 5-19% AND
- Be eligible for HMA therapy (very low riskthe possibility that something bad will happen participants are to be excluded)
- Locally or centrally confirmed IDH1 R132 C/G/H/L/S mutation