NIPH Clinical Trials Search

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)

Basic Information

Recruitment status	Recruiting
Health condition(s) or Problem(s) studied	hypomethylating agent (HMA) naive myelodysplastic syndromes (MDS) with an isocitrate dehydrogenase-1
Date of first enrollment	03/12/2024
Target sample size	7
Countries of recruitment	United States of America,Japan,France,Japan,Spain,Japan,Netherland,Japan,Germany,Japan,Italy,Japan,Australia,Japan,United Kingdom,Japan
Study type	Interventional
Intervention(s)	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.

Outcome(s)

Primary Outcome	CR+PR at 4 months as per IWG 2006 criteria
Secondary Outcome	Time to CR + PR as per IWG 2006 criteria Transfusion independence rate AML transformation rate Number of participants going to transplant

Key inclusion & exclusion criteria

Age minimum	>= 18age old
Age maximum	Not applicable
Gender	Both
Include 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
Exclude 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.