A Phase II Open-label Study of Olutasidenib Post-transplant Maintenance Therapy for Patients With IDH1-mutated Myeloid Malignancies

NCT06668584 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 2024-0522NCI-2025-00104
• Study Type: interventional
• Target: 31
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical research study is to learn about the safety and tolerability of giving olutasidenib to patients with IDH1-mutated myeloid malignancies as maintenance therapy after they receive a stem cell transplant.

Conditions

Myeloid Malignancies

Outcome Measures

Primary Outcomes (1)

• Safety and Adverse Events (AEs)

  Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0

  Through study completion; an average of 1 year

Study Arms (1)

Olutasidenib Maintenance Therapy

EXPERIMENTAL

Starting 30-120 days post-stem cell infusion, patients receive olutasidenib PO BID on days 1-28 of each cycle. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity. Patients also undergo blood sample collection throughout the study. Additionally, patients may undergo bone marrow aspiration and biopsy, ECHO/MUGA scan and chest x-ray at screening.

Drug: Olutasidenib

Interventions

Olutasidenib DRUG

Given by mouth

Olutasidenib Maintenance Therapy

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of AML, MDS, MPN, or CMML according to World Health Organization (WHO) classification that underwent first or second alloSCT regardless of stem cell source, donor type/match, conditioning regimen, or GVHD prophylaxis and is at least 30 days post stem cell transplant until day 120.
• Evdence of an IDH1 mutation presence by next generation sequencing panel at original diagnosis.
• Evidence of engraftment by Absolute neutrophil count (ANC) \>/= 1.0x 109/L without daily use of myeloid growth factor (G-CSF) for at least 7 days; and Platelet \>/= 30 x 109/L with out platelet transfusion within 1 week.
• Patient in morphologic remission with Day +30 bone marrow \<5% blast. Will include MRD positive or negative.
• Age 18 to 75 years old.
• ECOG performance status of 0, 1, 2. Or KPS above 70.
• Creatinine clearance greater or equal to 40cc/min as defined by the Cockcroft-Gault Equation.
• Males (mL/min): (140-age) \*IBW (kg) / 72\*(serum creatinine(mg/dl)) Females (mL/min): 0.85\*(140-age) \*IBW (kg) / 72\*(serum creatinine(mg/dl)).
• Serum bilirubin \</= 1.5 x upper limit of normal (ULN) except in subjects with Gilbert's Syndrome in whom total bilirubin must be \</= 3.0 mg/dl. Aspartate transaminase (AST) or alanine transaminase (ALT) \</= 2.5 x ULN. Alkaline phoshatase \</=2.5 x ULN.
• No active bleeding.
• No clinical evidence of life-threatening infection.
• Capable of understanding the investigational nature, potential risks, and benefits of the study, and able to provide valid informed consent.
• Negative serum or urine pregancy test for wome with reproductive potential at screening.
• Female participants of non-childbearing potential must meet at least one of the following criteria:
• Postmenopausal (no menses in greater than or equal to 12 consecutive months).

You may not qualify if:

• Use of any of the following after transplantation and prior to starting study therapy. Anti-leukemic agents given as post-transplant maintenance therapy (e.g., subcutaneous, or oral 5-azacitidine or FL T3 inhibitors for maintenance)
• Overall grade II-IV aGVHD. However, upon complete resolution of aGVHD-related symptoms with grade 1 or 0 overall clinical grade would be appropriate to enroll at that time. Patients may be eligible for enrollment if they are on prednisone 0.5 mg/kg daily dose or lower, tacrolimus, sirolimus, and/or ruxolitinib.
• cGVHD, moderate or servere by NIH criteria.
• Active uncontrolled systemic fungal, bacterial, or viral infection. However, patients receiving anti-micobial agents including antibiotics, antiviral and antifungal therapies are allowed if the infection is controlled, and the patient is hemodynamically stable.
• Known active viral infection with Human Immunodeficiency Virus (HIV), Hepatitis B Virus (HBV), or Hepatitis C Virus (HCV).
• Patients with known active hepatitis B virus (HBV) infection will be excluded because of potential effects on immune function and/or drug interactions. However, if a patient has HBV history with an undetectable HBV load by polymerase chain reaction (PCR), no liver-related complications, and is on definitive HBV therapy that is not contraindicated in this study, then they would be eligible for study.
• Patients with known active hepatitis C virus (HCV) infection will be excluded because of potential effects on immune function and/or drug interactions. However, if a patient with a history of HCV infection has received definitive therapy (and is now HCV viral load negative), or if a patient has a reactive HCV antibody test but has an undetectable viral load by PCR, then they would be eligible.
• Patients with known active HIV infection will be excluded out of concern for the drug-drug interaction with venetoclax and highly active antiretroviral therapy (HART)
• QT prolongation of corrected QTcF interval (QTc) \> 480 milliseconds
• Any other condition that would, in the Investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns with clinical study procedures.
• Patients with cognitive impairments or psychiatric disorders that can interfere with safety or with obtaining informed consent of compliance with study procedures.

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead

Study Sites (1)

The University of Texas M. D. Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Related Links

• MD Anderson Cancer Center Website

MeSH Terms

Interventions

olutasidenib

Study Officials

• Jeremy Ramdial, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jeremy Ramdial, MD

CONTACT

(713) 745-0146; jlramdial@mdanderson.org

Study Design

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

Study Record Dates

• First Submitted: October 30, 2024
• First Posted: October 31, 2024
• Study Start: December 31, 2024
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2029
• Last Updated: March 4, 2026

Record last verified: 2026-03

Locations

US (1)