A Phase 3, multicenter, open label, randomized, non-comparative two-arm study of ivosidenib monotherapy (IVO) and azacitidine monotherapy (AZA) in adult patients with hypomethylating agent (HMA) naive myelodysplastic syndromes (MDS) with an IDH1 mutation (PyramIDH study)

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) - Ivosidenib (IVO) monotherapy and azacitidine (AZA) monotherapy in patients with hypomethylating agent (HMA) naive myelodysplastic syndromes (MDS) with an IDH1 mutation

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2051240209

Enrollment

Registered

2024-12-10

Start date

2024-12-03

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

hypomethylating agent (HMA) naive myelodysplastic syndromes (MDS) with an isocitrate dehydrogenase-1 Myelodysplastic syndromes

Interventions

Ivosidenib 500 mg (2 tablets of 250 mg) daily every day or azacitidine 75 mg/m2/day every 4 weeks for 1 week, subcutaneously or intravenously.

Oral administration,Subcutaneous or intravenous administration

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Diagnosis of HMA naive IDH1 R132 mutated MDS defined according to World Health Organization (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

Exclusion criteria

Exclusion criteria: Have an active malignancy requiring anticancer treatment at the time of randomization. However, participants with the following history/concurrent conditions or similar indolent cancer are allowed to participate in the study: a. Basal or squamous cell carcinoma of the skin. b. Carcinoma in situ of the cervix. c. Carcinoma in situ of the breast. d. Incidental histologic finding of prostate cancer. 20% more blasts by morphology or immunohistochemistry on screening bone marrow aspirate. Have significant active cardiac disease within 6 months prior to the start of IMP in the judgment of the Investigator, including New York Heart Association (NYHA) Class III or IV congestive heart failure, myocardial infarction; unstable angina,and/or stroke. Have left ventricular ejection fraction (LVEF) less than 40% by echocardiogram (ECHO) scan (or by other methods according to institutional practice) obtained within 28 days prior to the start of IMP.

Design outcomes

Primary

Measure	Time frame
CR+PR at 4 months as per IWG 2006 criteria	—

Secondary

Measure	Time frame
Time to CR + PR as per IWG 2006 criteria Transfusion independence rate AML transformation rate Number of participants going to transplant	—

Countries

Australia, France, Germany, Italy, Japan, Netherland, Spain, United Kingdom, United States of America

Contacts

Public ContactICTR-Japan

Nihon Servier Company Limited

clinicaltrials.jpn@servier.com+81-3-4520-2350

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026