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)

JRCT ID: jRCT2051240209

Registered date:10/12/2024

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.

Related Information

• Primary Sponsor: Patel Prapti