Recruiting
Phase 1
Phase 2

Bleximenib

Sponsor:

Janssen Research & Development, LLC

Code:

NCT04811560

Conditions

Acute Leukemia

Eligibility Criteria

Sex: All

Age: 2+

Healthy Volunteers: Not accepted

Interventions

Bleximenib

Study Details

Brief summary:

The purpose of this study is to determine the recommended Phase 2 dose(s) (RP2D\[s\]) of bleximenib in phase 1 Part 1 (Dose Escalation) and to determine the safety and tolerability at RP2D in Phase 1 Part 2 (Dose expansion). The purpose of the Phase 2 part of the study is to evaluate the efficacy of bleximenib at the RP2D.

Conditions

Acute Leukemia

Study ID

NCT04811560

Start date

May 19, 2021

Status verified date

Aug, 2026

Completion date

Sep 22, 2030

Anticipated

Primary completion date

Feb 26, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2+

Healthy Volunteers: Not accepted

Inclusion Criteria:

Phase 1:

  • Age 2 years to less than (<) 18 years of age (pediatric cohort only), all other cohorts 18 years and above
  • Relapsed or refractory (R/R) acute leukemia and has exhausted, or is ineligible for, available therapeutic options
  • Acute leukemia harboring histone-lysine N-methyltransferase 2A (KMT2A), nucleophosmin 1 gene (NPM1) or nucleoporin 98 gene or nucleoporin 214 gene (NUP98 or NUP214) alterations

Phase: 2

  • Participants greater than 18 years are eligible
  • Must have had an initial diagnosis of acute myeloid leukemia (AML) per the WHO 2022 classification criteria and have relapsed/refractory disease
  • AML harboring KMT2A-r (gene rearrangement/translocation) or NPM1 mutations only

For Both Phase 1 and 2:

  • Pretreatment clinical laboratory values meeting the following criteria: (a) Hematology: white blood cell (WBC) count <= 20\*10\^9/liter (L) and (b) renal function; For adult participants, estimated or measured glomerular filtration rate >= 30 milliliter per minute (mL/min) per four variable MDRD equation. For pediatric participants an estimated or measured glomerular filtration rate >=40 mL/min per the CKiD (Chronic Kidney Disease in Children) Schwartz formula
  • Eastern Cooperative Oncology Group (ECOG) performance status grade of 0, 1 or 2. Pediatric participants only: Performance status >=70 by Lansky scale (for participants < 16 years of age) or >=70 Karnofsky scale (for participants >=16 years of age)
  • A female of childbearing potential must have a negative highly sensitive serum beta-human chorionic gonadotropin at screening and within 48 hours prior to the first dose of study treatment
  • Participant must agree to all protocol required contraception requirements and avoid sperm or egg donations or freezing for future reproductive use while on study and for 90 days (males) or 6 months (females) after the last dose of study treatment

Exclusion Criteria:

  • Acute promyelocytic leukemia, diagnosis of Down syndrome associated leukemia or juvenile myelomonocytic leukemia according to World Health Organization (WHO) 2016 criteria
  • Active central nervous system (CNS) disease
  • Prior solid organ transplantation
  • QTc according to Fridericia's formula (QTcF) for males >= 450 millisecond (msec) or for females >= 470 msec. Participants with a family history of Long QT syndrome are excluded
  • Exclusion criteria related to stem cell transplant: a. Received prior treatment with allogenic bone marrow or stem cell transplant <=3 months before the first dose of study treatment ; b. Has evidence of graft versus host disease; c. Received donor lymphocyte infusion <=1 month before the first dose of study treatment; d. Requires immunosuppressant therapy (exception: daily doses <=10 milligrams (mg) prednisone or equivalent are allowed for adrenal replacement)
  • Prior cancer immunotherapy within 4 weeks prior to enrollment or blinatumomab within 2 weeks prior to enrollment. Additional prior cancer therapies must not be given within 4 weeks prior to enrollment or 5 half-lives of the agent (whichever is shorter)

Study Design

Enrollment

420 participants

Anticipated

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Bleximenib

Participants in Phase 1 Part 1 (dose escalation) will receive bleximenib orally. The dose levels will be escalated based on the dose limiting toxicities (DLT) evaluation by Study Evaluation Team (SET) until the recommended Phase 2 Doses (RP2Ds) have been identified. Participants in Phase 1 Part 2 (dose expansion) will receive bleximenib orally at the RP2D(s) determined in Part 1. In Phase 2 participants will receive bleximenib at the RP2D to evaluate anti-leukemia activity and demonstrate acceptable safety at the RP2D(s).

Interventions

Bleximenib

Bleximenib is administered orally.

Primary outcome measure

  • Phase 1: Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability [ Time Frame: Up to 4 years and 9 months ]
  • Phase 1: Number of Participants with AEs by Severity [ Time Frame: Up to 4 years and 9 months ]
  • Phase 1: Part 1: Percentage of Participants with Dose-Limiting Toxicity (DLT) [ Time Frame: Up to 28 days Cycle 1 ]
  • Phase 2: Rate of Complete Remission or Complete Remission with Partial Hematologic Recovery (CR/CRh) [ Time Frame: Up to 4 years and 9 months ]

Central Contacts and Locations

Central contacts

Study Contact

844-434-4210Participate-In-This-Study1@its.jnj.com

Locations

City of Hope

Recruiting

Duarte, California, United States, 91010

University of California San Francisco

Recruiting

San Francisco, California, United States, 94143

UCSF Benioff Children's Hospital

Recruiting

San Francisco, California, United States, 94158

University of Chicago

Recruiting

Chicago, Illinois, United States, 60637

St Francis Hospital & Health Centers Indiana Blood and Marrow Transplantation

Recruiting

Indianapolis, Indiana, United States, 46237

Massachusetts General Hospital

Recruiting

Boston, Massachusetts, United States, 02114

Dana Farber Cancer Institute

Recruiting

Boston, Massachusetts, United States, 02115

Start Midwest

Recruiting

Grand Rapids, Michigan, United States, 49546

University of New Mexico

Recruiting

Albuquerque, New Mexico, United States, 87131

Roswell Park Comprehensive Cancer Center

Recruiting

Buffalo, New York, United States, 14263

NYU Langone Medical Center

Recruiting

New York, New York, United States, 10016

Montefiore Medical Center

Recruiting

The Bronx, New York, United States, 10467

Oregon Health And Science University

Recruiting

Portland, Oregon, United States, 97239

Maine Health

Recruiting

Providence, Rhode Island, United States, 02903

University of Tennessee Medical Center

Recruiting

Knoxville, Tennessee, United States, 37920

Baylor University Medical Center

Recruiting

Dallas, Texas, United States, 75246

Houston Methodist Hospital

Recruiting

Houston, Texas, United States, 77030-2740

MD Anderson

Recruiting

Houston, Texas, United States, 77030

San Antonio Methodist TX Transplant Physicians Group

Recruiting

San Antonio, Texas, United States, 78229

Virginia Commonwealth University

Recruiting

Richmond, Virginia, United States, 23298

Swedish Cancer Institute

Recruiting

Seattle, Washington, United States, 98104

Medical College of WI at Froedtert

Recruiting

Milwaukee, Wisconsin, United States, 53226

Vancouver Coastal Health

Recruiting

Vancouver, British Columbia, Canada, V5Z 1M9

Princess Margaret Cancer Centre University Health Network

Recruiting

Toronto, Ontario, Canada, M5G 1X6

Hopital Maisonneuve Rosemont

Recruiting

Montreal, Quebec, Canada, H1T 2M4

More Information

Sponsor

Janssen Research & Development, LLC

Last update posted

Aug 28, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Janssen Research & Development, LLC on 2026-08-28.