Relapsed or Refractory IDH1 Mutation-Positive Acute Myeloid LeukemiaPhase 2NCT07604064
A Clinical Trial of Olutasidenib in Patients With Acute Myeloid Leukemia
A Phase 2 study testing Olutasidenib in people with Relapsed or Refractory IDH1 Mutation-Positive Acute Myeloid Leukemia, run by Kissei Pharmaceutical Co., Ltd. at 1 site in Japan.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Relapsed or Refractory IDH1 Mutation-Positive Acute Myeloid Leukemia
- What is being tested?
- Olutasidenib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteJapan
- Study dates
- Jun 2026to Sep 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Japan1 site, 1 recruiting
- Recruiting
Research Site
Tokyo and Other Japanese Cities
About this study
Official title: 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.
- Being tested
- Olutasidenib
- Medical subject headings
- Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OlutasidenibDrug
Olutasidenib: Oral administration
| Study group | Role | What they receive |
|---|---|---|
| Olutasidenib | Experimental | Olutasidenib will be administered orally twice daily under fasting condition. Receives: Drug: Olutasidenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From the start of IMP administration to 28 days after the final dose of the IMP
Secondary outcomes (5)
CR/CRh rate
The number and proportion of patients who achieve CR or CRh
Measured: 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
Measured: 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
Measured: 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
Measured: Through study completion, approximately up to 3 years
Overall survival (OS)
The duration from the start of IMP administration to death for any reason
Measured: Through study completion, approximately up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2026
- Study startJun 2026 (estimated)
- Primary completionSep 2030 (estimated)
- Study completionSep 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed or Refractory IDH1 Mutation-Positive Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kissei Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yumi Ikezaki, Study director, Kissei Pharmaceutical Co., Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07604064
- Sponsor's protocol ID
- OLT1201
- Other IDs
- jRCT2051260041
- Status last verified
- May 2026
- First submitted
- 18 May 2026
- Last updated
- 27 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: * 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.
Study at a glance
From the registry record, last updated by the sponsor on 27 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Clinical Trial of Olutasidenib in Patients With Relapsed or Refractory IDH1 Mutation-Positive Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT07604064
- Acronym / sponsor ID
- OLT1201
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 3 participants (estimated)
- Lead sponsor
- Kissei Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- Jun 2026 (estimated)
- Primary completion
- Sep 2030 (estimated)
- Study completion
- Sep 2030 (estimated)
- First posted
- 22 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Olutasidenib | Experimental | Drug: OlutasidenibOlutasidenib will be administered orally twice daily under fasting condition. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OlutasidenibOlutasidenib: Oral administration | Drug | Olutasidenib |
Primary outcome
Incidence of adverse events and adverse drug reactions
Time frame: From the start of IMP administration to 28 days after the final dose of the IMP
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.
5 secondary outcomes
CR/CRh rate
Time frame: Through study completion, approximately up to 3 years
Duration of CR/CRh
Time frame: Through study completion, approximately up to 3 years
Time to CR/CRh
Time frame: Through study completion, approximately up to 3 years
Transfusion independence
Time frame: Through study completion, approximately up to 3 years
Overall survival (OS)
Time frame: Through study completion, approximately up to 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 May 2026.
- Individual participant dataSharing planned: Yes. * 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.
Related trials for Relapsed or Refractory IDH1 Mutation-Positive Acute Myeloid Leukemia
RecruitingPhase 1
Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation
Relapsed or Refractory Acute Myeloid Leukemia (AML), Untreated AML
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Yumi Ikezaki study director
Kissei Pharmaceutical Co., Ltd.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Olutasidenib in people with Relapsed or Refractory IDH1 Mutation-Positive Acute Myeloid Leukemia, run by Kissei Pharmaceutical Co., Ltd. at 1 site in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 27 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kissei Pharmaceutical Co., Ltd (Email only, rinsyousiken@pharm.kissei.co.jp), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events and adverse drug reactions (time frame: From the start of IMP administration to 28 days after the final dose of the IMP). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 May 2026.