Olutasidenib Single Plus Combo Therapy in IDH1mut AML After Induction and Consolidation

NCT07130695 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: MCC-25-22096
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

Treatment with olutasidenib for isocitrate dehydrogenase 1 (IDH1) mutant acute myeloid leukemia (AML) after completion of traditional intensive induction/consolidation is likely to be safe, tolerable, and may provide clinical benefit in terms of maintenance of remission and perhaps improvement in survival.

Conditions

Acute Myeloid Leukemia

Keywords

Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (2)

• Assess the feasibility of olutasidenib after upfront acute myeloid leukemia (AML) therapy with intensive induction and/or consolidation in IDH1 mutant AML.

  Feasibility defined as the number of participants with 75% protocol treatment compliance for a duration of at least 4 cycles (to include protocol defined dose delays), or until disease relapse or allogeneic hematopoietic stem cell transplant (alloHCT).

  Up to 2 years

• Assess the tolerability of olutasidenib after upfront AML therapy with intensive induction and/or consolidation in IDH1 mutant AML

  Incidence of grade ≥4 adverse events (AEs) attributable to study drug for duration of treatment on study

  Up to 2 years

Secondary Outcomes (5)

• Measure progression free survival

  Up to 2 years following end of treatment

• Estimate overall survival (OS)

  Up to 2 years following end of treatment

• Assess mean residual disease (MRD) negativity rates by both PCR based and flow cytometric methodologies

  Baseline, Cycle 3 Day 15, and End of Treatment (Each cycle is 28 days)

• Estimate rates of ability to proceed with transplant given maintenance opportunities.

  Up to 2 years

• Characterize quality of life (QOL) metrics by SF 36

  Cycle 1Day 1, Cycle 2 Day 1, Cycle 4 day 1, and End of Treatment (EOT) (Each cycle is 28 days)

Study Arms (1)

Olutasidenib Investigational Agent Administration

EXPERIMENTAL

150 mg by mouth twice daily.

Drug: Olutasidenib Investigational Agent Administration

Interventions

Olutasidenib Investigational Agent Administration DRUG

Twice daily olutasidenib maintenance therapy

Olutasidenib Investigational Agent Administration

Eligibility Criteria

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

You may qualify if:

• Histologically or cytologically confirmed non-acute promyelocytic isocitrate dehydrogenase (1 IDH1) mutant acute myeloid leukemia (AML). IDH1 mutation may be identified by NGS or PCR based methods and identified at time of diagnosis or any other time point prior to enrollment.
• Completed induction and/or consolidation intended as per treating physician to reach complete response (CR),complete response with partial hematologic recovery (CRh), or complete response with incomplete hematologic recovery (CRi), or morphologic leukemia free state (MLFS) at time of study enrollment Patients must be within 90 days of their last cycle of upfront therapy.
• Age ≥18 years
• Calculated creatinine clearance (by Cockroft-Gault) ≥30 mL/min
• Total bilirubin ≤2 × upper limit of normal (ULN) Note: patients with Gilbert's syndrome may be included if total bilirubin is ≤3 × ULN and direct bilirubin is ≤2 × ULN
• Serum aspartate aminotransferase/ alanine aminotransferase (AST/ALT) ≤3 × ULN
• Eastern Cooperative Oncology Group (ECOG) 0, 1, or 2 or KPS \>50%
• Able to take oral medications
• Women of childbearing potential must consent to effective contraception during study treatment and at least 6 months following the last dose. Effective methods of contraception include oral or injectable hormonal birth control, intrauterine device (IUD), and double- barrier methods. (ie, combination of male condom with either cap, diaphragm or sponge with spermicide)
• Male participants who are sexually active with a woman of childbearing potential and who have not had vasectomies must be willing to use a barrier method of contraception and refrain from sperm donation from initial study drug until 90 days after last dose of study drug.

You may not qualify if:

• History of hypersensitivity or allergic reaction to olutasidenib or its components
• Corrected Q-T interval (QTc) (Fredericia calculation) \> 450 ms (after corrective action is taken)
• History of Torsades de Pointes
• Any gastrointestinal condition thought by the treating investigator to impair oral absorption of medication
• Stem cell transplant eligible and planned within 60 days of study start date in the opinion of the treating investigator
• Uncontrolled intercurrent illness or infection (those with controlled human immunodeficiency virus (HIV), hepatitis, or other chronic infections are eligible)
• Female participants who are pregnant or intend to donate eggs during the study or for 6 months after receiving their last dose of study drug
• Nursing women, women of childbearing potential with positive pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception. (Appropriate method(s) of contraception include oral or injectable hormonal birth control, IUD, and double-barrier methods)
• Male participants who intend to donate sperm during the course of this study or for 3 months after last dose
• Participants receiving, or are expected to require during the study, any concomitant medications that may interfere with efficacy, metabolism, or safety of the investigational agent, including drugs known to cause QT prolongation. for which drug interactions with olutasidenib would be prohibitory
• Concurrent chemotherapy for non-AML malignancy that is expected to interfere with the efficacy, metabolism, or safety of the agent under investigation
• Received non-intensive upfront therapy including hypomethylating agents (HMA) /Venetoclax based
• Currently receiving other targeted therapies or AML directed therapies, including but not limited to other IDH1 or IDH2 inhibitors, FMS-like tyrosine kinase 3 (FLT3) inhibitors, B-cell lymphoma 2 (BCL-2) inhibitors, menin inhibitors
• Other investigational agents in another clinical trial within 4 weeks prior to enrollment
• Systemic corticosteroids above physiologic replacement doses (10mg/day prednisone or equivalent), unless used to tread IDH differentiation syndrome or as part of a pre-specified protocol exception

Sponsors & Collaborators

• Virginia Commonwealth University: lead
• Rigel Pharmaceuticals: collaborator

Study Sites (1)

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Keri Maher, DO

  Virginia Commonwealth University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Massey IIT Research Operations

CONTACT

804-628-6430; masseyepd@vcu.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 7, 2025
• First Posted: August 19, 2025
• Study Start: February 2, 2026
• Primary Completion (Estimated): October 31, 2028
• Study Completion (Estimated): October 31, 2030
• Last Updated: February 6, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)