Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation

Launched by INSTITUT DE RECHERCHES INTERNATIONALES SERVIER · Jun 14, 2024

Keywords

ClinConnect Summary

This clinical trial is studying two different treatment options for patients with myelodysplastic syndromes (MDS) who have a specific genetic mutation called IDH1. The trial is for individuals who have not previously received treatment with a type of medication known as hypomethylating agents. Participants will be randomly assigned to receive either ivosidenib (IVO), taken daily for 28 days, or azacitidine (AZA), which is given for the first 7 days of each 28-day cycle. The study aims to see how well these treatments work and to monitor the safety of each option.

To be eligible for this trial, participants must have a confirmed diagnosis of MDS with the IDH1 mutation and meet certain blood count criteria. This includes having specific low blood cell counts and a certain percentage of immature cells (blasts) in their blood or bone marrow. Throughout the trial, participants will have regular check-ups, which may include physical exams and blood tests, to monitor their health and response to treatment. It's important to note that individuals who have previously received other MDS treatments or have a high percentage of immature cells are not eligible for this trial.

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ALL

Eligibility criteria

• Inclusion Criteria:
• * Diagnosis 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 platelets/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 risk participants are to be excluded)
• • Locally or centrally confirmed IDH1 R132 C/G/H/L/S mutation
• Exclusion Criteria:
• • Received prior anticancer/disease modifying treatment for MDS (including HMA's, cytotoxic chemotherapy, investigational agents, bcl-2 inhibitor based-regimens, hematopoietic stem cell transplant (HSCT), IDH1 inhibitors). For LR-MDS patients, prior treatment with growth factors, luspatercept, lenalidomide, and imetelstat are allowed.
• • \>20% blasts by morphology or immunohistochemistry on screening bone marrow aspirate/biopsy