A Clinical Trial of Olutasidenib in Patients With Acute Myeloid Leukemia

A Phase II Clinical Trial of Olutasidenib in Patients With Relapsed or Refractory IDH1 Mutation-Positive Acute Myeloid Leukemia

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.

Study Overview

• Status: Recruiting
• Conditions: Relapsed or Refractory IDH1 Mutation-Positive Acute Myeloid Leukemia
• Intervention / Treatment: Drug: Olutasidenib

• Study Type: Interventional
• Enrollment (Estimated): 3
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Kissei Pharmaceutical Co., Ltd; Phone Number: Email only; Email: rinsyousiken@pharm.kissei.co.jp

Study Locations

• Japan
  • Tokyo and Other Japanese Cities, Japan
    • Recruiting
    • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Olutasidenib; Olutasidenib will be administered orally twice daily under fasting condition.	Drug: Olutasidenib; Olutasidenib: Oral administration

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
CR/CRh rate	The number and proportion of patients who achieve CR or CRh	Through study completion, approximately up to 3 years
Duration of CR/CRh	The duration from the achievement of the first CR or CRh to relapse or death, whichever occurs first	Through study completion, approximately up to 3 years
Time to CR/CRh	The duration from the start of IMP administration to the achievement of CR or CRh	Through study completion, approximately up to 3 years
Transfusion independence	The number and proportion of patients who have been transfusion-free for 28 days and 56 days after the start of IMP	Through study completion, approximately up to 3 years
Overall survival (OS)	The duration from the start of IMP administration to death for any reason	Through study completion, approximately up to 3 years

Collaborators and Investigators

• Sponsor: Kissei Pharmaceutical Co., Ltd.
• Investigators: Study Director: Yumi Ikezaki, Kissei Pharmaceutical Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): September 1, 2030
• Study Completion (Estimated): September 1, 2030

Study Registration Dates

• First Submitted: May 18, 2026
• First Submitted That Met QC Criteria: May 18, 2026
• First Posted (Actual): May 22, 2026

Study Record Updates

• Last Update Posted (Actual): May 27, 2026
• Last Update Submitted That Met QC Criteria: May 22, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Disease Attributes; Pathological Conditions, Signs and Symptoms; Recurrence; olutasidenib
• Other Study ID Numbers: OLT1201; jRCT2051260041 (Registry Identifier: jRCT)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: The Individual Patient Data (IPD) are available upon reasonable request and with permission of Kissei Pharmaceutical Co., Ltd.; For a data sharing request for IPD, please contact Kissei Pharmaceutical at rinsyousiken@pharm.kissei.co.jp.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No