Olutasidenib for relapsed or refractory IDH1-mutant AML in Japanese patients
Official title: A Phase II Clinical Trial of Olutasidenib in Patients With Relapsed or Refractory IDH1 Mutation-Positive Acute Myeloid Leukemia
Run by Kissei Pharmaceutical Co., Ltd. · NCT07604064 · Data updated 2026-11-12
This study tests whether taking olutasidenib by mouth twice daily for one 28-day cycle is safe and shows activity in Japanese adults with relapsed or refractory IDH1-mutant AML.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 3 (estimated) |
| Sponsor | Kissei Pharmaceutical Co., Ltd. Industry-sponsored |
| Locations | 1 site (Tokyo and Other Japanese Cities) |
| Status | Recruiting |
| Started | June 2026 |
| Main results expected | September 2030 |
| Study ends | September 2030 |
| Registry record updated | May 27, 2026 |
| Trial ID | NCT07604064 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Adults in Japan with relapsed or refractory IDH1-mutant AML, confirmed by central testing, who can provide informed consent and meet criteria including WHO 2022 AML diagnosis (excluding acute promyelocytic leukemia with t(15;17)).
✕Probably not a fit
Patients with IDH2 mutations, prior IDH2 inhibitor exposure, or those intolerant to IDH1 inhibitors may not benefit from this treatment.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is a multicenter, single-arm, open-label Phase 2 trial enrolling Japanese patients with relapsed or refractory IDH1-mutant acute myeloid leukemia. Participants will receive olutasidenib orally twice daily after fasting for one 28-day cycle, with safety, efficacy, pharmacokinetics, and pharmacodynamics assessed. Central confirmation of IDH1 mutation is required, and prior treatments including stem cell transplantation are allowed. Exclusion criteria include IDH2 mutations or prior IDH2 inhibitor exposure. The study aims to characterize how the drug behaves in this population and whether it produces clinical benefit.
Why it matters
Potential benefit: If successful, participants may experience reduced disease burden or prolonged remission and gain information that could support future treatments for IDH1-mutant AML.
How similar studies have gone: Previous studies in IDH1-mutant AML have shown activity with IDH1 inhibitors in selected populations, but this Japanese cohort represents a targeted, ethnically specific assessment.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Japanese patients who personally provide written informed consent to participate in this clinical trial * Patients with a confirmed diagnosis of AML based on WHO classification (2022 edition) (except acute promyelocytic leukemia with t (15:17) translocation) * Patients with relapsed or refractory AML who may or may not have undergone allogeneic hematopoietic stem cell transplantation. * Patients with IDH1 gene mutation confirmed by central confirmation after relapse or refractoriness Exclusion Criteria: * Patients with IDH2 mutations or patients with a history of IDH2 inhibitor treatment * Patients who are intolerant to IDH1 inhibitors * Patients who are deemed inappropriate for the clinical trial by the investigator or sub-investigator
Where it's running
-
Research Site
Tokyo and Other Japanese Cities, Japan Recruiting
Study contacts
- Study coordinator: Kissei Pharmaceutical Co., Ltd
- Email: rinsyousiken@pharm.kissei.co.jp
- Phone: Email only
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.