A Phase II Clinical Trial of Olutasidenib in Patients With Acute Myeloid Leukemia

NCT07604064 | Recruiting Phase 2

• 2 other identifiers: OLT1201jRCT2051260041
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

This clinical trial is a multicenter, single-arm, open-label study to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of olutasidenib administered orally twice daily under fasting conditions for one cycle of 28 days in at least 3 Japanese patients with relapsed or refractory IDH1 mutation-positive AML.

Conditions

Relapsed or Refractory IDH1 Mutation-Positive Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse events and adverse drug reactions

  The number of events, number of patients, and incidence will be presented for all events, Grade 3 or higher events, Grade 4 or higher events, events resulting in death, serious events excluding death, events resulting in drug withdrawal, and events resulting in drug interruption.

  From the start of IMP administration to 28 days after the final dose of the IMP

Secondary Outcomes (5)

• CR/CRh rate

  Through study completion, approximately up to 3 years

• Duration of CR/CRh

  Through study completion, approximately up to 3 years

• Time to CR/CRh

  Through study completion, approximately up to 3 years

• Transfusion independence

  Through study completion, approximately up to 3 years

• Overall survival (OS)

  Through study completion, approximately up to 3 years

Study Arms (1)

Olutasidenib

EXPERIMENTAL

Olutasidenib will be administered orally twice daily under fasting condition.

Drug: Olutasidenib

Interventions

Olutasidenib DRUG

Olutasidenib: Oral administration

Olutasidenib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Kissei Pharmaceutical Co., Ltd.: lead

Study Sites (1)

Research Site

Tokyo and Other Japanese Cities, Japan

RECRUITING

MeSH Terms

Conditions

Recurrence

Interventions

olutasidenib

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Yumi Ikezaki

  Kissei Pharmaceutical Co., Ltd.

  STUDY DIRECTOR

Central Study Contacts

Kissei Pharmaceutical Co., Ltd

CONTACT

Email only; rinsyousiken@pharm.kissei.co.jp

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 18, 2026
• First Posted: May 22, 2026
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): September 1, 2030
• Study Completion (Estimated): September 1, 2030
• Last Updated: May 22, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share

Locations

JP (1)