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Treatment for Myelodysplastic Syndromes with IDH1 Mutation

A Phase 3, Multicenter, Open Label, Randomized, Non-comparative Two-arm Study of Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Adult Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an Isocitrate Dehydrogenase-1 (IDH1) Mutation (PyramIDH Study)

Phase 3 Interventional Servier · NCT06465953

This study is testing whether a new drug called ivosidenib can help people with myelodysplastic syndromes and an IDH1 mutation feel better compared to the standard treatment with azacitidine.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment48 (estimated)
Ages18 Years and up
SexAll
SponsorServier Industry-sponsored
Drugs / interventionschemotherapy
Locations62 sites (Denver, Colorado and 61 other locations)
Trial IDNCT06465953 on ClinicalTrials.gov

What this trial studies

This clinical trial enrolls patients with myelodysplastic syndromes (MDS) who have an IDH1 mutation and have not previously received hypomethylating agents. Participants will be randomly assigned to receive either ivosidenib (IVO) or azacitidine (AZA) as monotherapy. IVO will be taken daily for 28-day cycles, while AZA will be administered for the first 7 days of each cycle. The study includes regular visits for monitoring and assessments, including blood tests and bone marrow evaluations, to determine the effectiveness and safety of the treatments.

Who should consider this trial

Good fit: Ideal candidates are patients diagnosed with IDH1 mutated MDS who have not received prior treatment with hypomethylating agents.

Not a fit: Patients with very low-risk MDS or those who have previously received anticancer treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new effective treatment option for patients with MDS and IDH1 mutations.

How similar studies have performed: Other studies have shown promise with similar approaches targeting IDH1 mutations, indicating potential for success.

Eligibility criteria

Show full inclusion / exclusion 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

Where this trial is running

Denver, Colorado and 61 other locations

+12 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Hypomethylating Agent Naive Myelodysplastic SyndromesMyelodysplastic Syndromes
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.