NCT06707493

Ivosidenib as Post-HSCT Maintenance for AML

A Randomized, Placebo-Controlled Phase 2 Study of IDH1 Inhibition Using Ivosidenib as Maintenance Therapy for IDH1-mutant Acute Myeloid Leukemia Following Allogeneic Stem Cell Transplantation

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 75 (estimated)
• Sponsor: Massachusetts General Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2 study of the study drug, ivosidenib (a mutant IDH1 inhibitor), compared to placebo, given to patients with IDH1-mutant acute myeloid leukemia (AML) after hematopoietic stem cell transplantation (HCT).

Detailed description

This is a prospective, placebo-controlled, randomized, single-blinded, multi-center, phase II study of the mutant IDH1 inhibitor, ivosidenib, compared to placebo in participants with AML after HCT. This study is examining whether or not ivosidenib is beneficial as an agent to prevent the relapse of IDH1-mutated acute myeloid leukemia after hematopoietic stem cell transplantation. The U.S. Food and Drug Administration (FDA) has not approved ivosidenib for this indication following HCT but it has been approved for other uses. The research study procedures include screening for eligibility and study treatment including evaluations and follow-up. The HCT and any standard treatment before and after the HCT is standard of care. The estimated length of participation in the study is 3.5 years from screening to the end of planned follow-up, including up to 24 months of study treatment. After the 24-month period, participants are followed for up to 12 additional months. It is expected that about 75 people will take part in this research study. Servier, a pharmaceutical company, is supporting this research study by providing ivosidenib/placebo and funding for research activities.

Conditions

• IDH1 Mutation
• Acute Myeloid Leukemia (AML)
• Hematopoietic Stem Cell Transplant (HSCT)

Interventions

Type	Name	Description
DRUG	Ivosidenib	Ivosidenib tablets are supplied as 50 mg, 200 mg, and 250 mg strengths, to be taken orally.
DRUG	Placebo	Placebo tablets are taken orally.

Timeline

• Start date: 2026-01-16
• Primary completion: 2028-01-01
• Completion: 2030-01-01
• First posted: 2024-11-27
• Last updated: 2026-03-10

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study