Compares ivosidenib and azacitidine in people with IDH1-mutated myelodysplastic syndromes
Compares two treatments in adults with myelodysplastic syndromes who have an IDH1 gene change.
Plain-language title generated from the registry record
Registry title: Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation
Key facts
- Where
- Ancona, Bologna, Florence, Pavia, Rome, Rozzano, and Turin, ItalySee the 7 sites
- Phase
- Phase 3
Where in Italy
- Azienda Ospedaliero-Universitaria delle MarcheAncona, ItalyTaking people
- Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere ScientificoBologna, ItalyTaking people
- Careggi University HospitalFlorence, ItalyTaking people
- Fondazione IRCCS Policlinico San MatteoPavia, ItalyTaking people
- Universita' Degli Studi Di Roma Tor VergataRome, ItalyTaking people
- Humanitas Mirasole S.p.A.Rozzano, ItalyTaking people
- Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di TorinoTurin, ItalyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Institut de Recherches Internationales Servier
- Trial code
- NCT06465953EU 2023-510155-37-00
- Record updated
- 3 September 2026
- Original description
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.Read full original descriptionShow less
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)
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.
Inclusion: 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: 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.