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

Recruiting now · Phase 3

Conditions studied: Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS), Myelodysplastic Syndromes (MDS)

In brief

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 dose 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 marrow aspirate, physical exam, echocardiogram (ECHO), electrocardiogram (ECG), blood and urine analysis, and questionnaires.

Key facts

Study ID

NCT06465953

Run by

Institut de Recherches Internationales Servier

People needed

48

Starts

2024-12-03

Expected to finish

2028-12-01

Last updated by the study team

2026-08-05

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Universitatsklinikum Dresden Carl Gustav Carus — Dresden, Germany (enrolling)
• Marien Hospital Duesseldorf — Düsseldorf, Germany (enrolling)
• University of Chicago, Duchossois Center for Advanced Medicine (DCAM) — Chicago, Illinois, United States (enrolling)
• Chu Brabois — Vandœuvre-lès-Nancy, France (enrolling)
• Massachusetts General Hospital — Boston, Massachusetts, United States (enrolling)
• Oncology Associates of Oregon — Eugene, Oregon, United States (enrolling)
• MSKCC — New York, New York, United States (enrolling)
• MD Anderson Cancer Centre — Houston, Texas, United States (enrolling)
• Hopital Saint Louis — Paris, France (enrolling)
• Institut Universitaire Du Cancer Toulouse-Oncopole — Toulouse, France (enrolling)
• Northern Health — Epping, Australia (enrolling)
• Ohio State University Comprehensive Cancer Center — Columbus, Ohio, United States (enrolling)
• Sir Charles Gairdner Hospital — Nedlands, Australia (enrolling)
• Calvary Mater Newcastle — Waratah, Australia (enrolling)
• Liga Paranaense de Combate ao Câncer - Hospital Erasto Gaertner — Curitiba, Brazil (enrolling)
• Centro de Pesquisa Clínica - Hospital Nove de Julho — São Paulo, Brazil (enrolling)
• Real E Benemérita Associação Portuguesa de São Paulo — São Paulo, Brazil (enrolling)
• Hospital das Clínicas da Faculdade de Medicina da USP — São Paulo, Brazil (enrolling)
• Sociedade Beneficente Israelita Brasileira Hospital Albert Einstein — São Paulo, Brazil (enrolling)
• Unc Lineberger Comprehensive Cancer Center — Chapel Hill, North Carolina, United States (enrolling)
• Monash Health — Clayton, Australia (enrolling)
• Chu Bordeaux, Hopital Du Haut Leveque — Pessac, France (enrolling)
• Chu Nantes-Hotel Dieu — Nantes, France (enrolling)
• Chu de Nice - Hôpital L'Archet 1 — Nice, France (enrolling)
• Universitaetsmedizin Goettingen (Umg) — Göttingen, Germany (enrolling)

Full record on ClinicalTrials.gov

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