Under construction – not a live service
Sign in
Taking people in BrazilPhase 3

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.

Key facts

Who runs it
Institut de Recherches Internationales Servier
Where
Curitiba and São Paulo, BrazilSee the 8 sitesAlso in 9 other countries, 55 sites
Registry title
Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation
Status
Recruiting
Phase
Phase 3
People taking part
48 people, planned
Started
3 Dec 2024
Expected to end
1 Dec 2028
Type of study
Interventional, 2 groups
Treatments tested
  • Ivosidenib
  • Azacitidine
Record updated
3 September 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for adults with myelodysplastic syndromes (MDS) who have an IDH1 gene change and have not been treated for MDS before. They get either ivosidenib or azacitidine.

Age
18 years or older
Sex
All sexes can join.
Disease stage
MDS under WHO criteria (5th edition) with 0-19% blasts. Moderate high, high or very high-risk MDS can join whatever the blood counts. Low or moderate low-risk MDS can join only with low blood counts caused by MDS, 5-19% blasts, and when HMA therapy is possible. Very low-risk MDS cannot join.
Earlier treatment
No earlier treatment for MDS that is meant to fight the disease, such as HMA drugs, chemotherapy, study drugs, bcl-2 inhibitor regimens, stem cell transplant or IDH1 inhibitors. In low-risk MDS, earlier growth factors, luspatercept, lenalidomide and imetelstat are allowed.
Biomarker or test result
An IDH1 R132 C/G/H/L/S mutation, confirmed locally or centrally.

Main reasons for not taking part

  • Earlier anticancer or disease-modifying treatment for MDS (with the exceptions listed above)
  • More than 20% blasts in the bone marrow at screening
  • Very low-risk MDS

The registry criteria, word for word

Show all 9 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • 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 in Brazil

Also in 9 other countries, 55 sites

  • Liga Paranaense de Combate ao Cancer - Hospital Erasto GaertnerCuritiba, BrazilContact: Institut de Recherches Internationales Servier (I.R.I.S.), Clinical Studies DepartmentTaking people
  • Casa de Saude Santa MarcelinaSão Paulo, BrazilContact: Institut de Recherches Internationales Servier (I.R.I.S.), Clinical Studies DepartmentTaking people
  • Centro de Pesquisa Clínica - Hospital Nove de JulhoSão Paulo, BrazilContact: Institut de Recherches Internationales Servier (I.R.I.S.), Clinical Studies DepartmentTaking people
  • Centro de Pesquisas Clínicas da Fundação Doutor Amaral CarvalhoSão Paulo, BrazilContact: Institut de Recherches Internationales Servier (I.R.I.S.), Clinical Studies DepartmentTaking people
  • Hospital das Clinicas da Faculdade de Medicina da USPSão Paulo, BrazilContact: Institut de Recherches Internationales Servier (I.R.I.S.), Clinical Studies DepartmentTaking people
  • Instituto Nacional do CâncerSão Paulo, BrazilContact: Institut de Recherches Internationales Servier (I.R.I.S.), Clinical Studies DepartmentNot yet recruiting
  • Real E Benemerita Associacao Portuguesa de Sao PauloSão Paulo, BrazilContact: Institut de Recherches Internationales Servier (I.R.I.S.), Clinical Studies DepartmentTaking people
  • Sociedade Beneficente Israelita Brasileira Hospital Albert EinsteinSão Paulo, BrazilContact: Institut de Recherches Internationales Servier (I.R.I.S.), Clinical Studies DepartmentTaking people

About the trial

What the trial wants to find out

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.

The 2 groups in the trial

  • Ivosidenib monotherapyTreatment being tested

    Treatment: Ivosidenib

  • Azacitidine monotherapyTreatment being tested

    Treatment: Azacitidine

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
Official title
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)
Plain-language title
Compares ivosidenib and azacitidine in people with IDH1-mutated myelodysplastic syndromesGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 12 November 2026.
Record updated
3 September 2026