NCT04811560 · Janssen Research & Development, LLC
A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia (cAMeLot-1)
(cAMeLot-1)
What this study is about
The purpose of this study is to determine the recommended Phase 2 dose(s) (RP2D\[s\]) of bleximenib in phase 1 Part 1 (gradually increasing doses) and to determine the safety and how well patients handle the treatment at RP2D in Phase 1 Part 2 (Dose expansion). The purpose of the Phase 2 part of the study is to evaluate the effectiveness of bleximenib at the RP2D.
View original scientific description
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.
Interventions
DRUG
Bleximenib
Bleximenib is administered orally.
Primary outcome measures
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
An AE is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the pharmaceutical/biological agent under study.
Phase 1: Number of Participants with AEs by Severity
Time frame: Up to 4 years and 9 months
Severity will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0. Severity scale ranges from Grade 1 (Mild) to Grade 5 (Death). Grade 1= Mild, Grade 2= Moderate, Grade 3= Severe, Grade 4= Life-threatening and Grade 5= Death related to adverse event.
Phase 1: Part 1: Percentage of Participants with Dose-Limiting Toxicity (DLT)
Time frame: Up to 28 days Cycle 1
Percentage of participants with DLT will be assessed accordingly to national cancer institute common terminology criteria for adverse events (NCI-CTCAE) version 5.
Phase 2: Rate of Complete Remission or Complete Remission with Partial Hematologic Recovery (CR/CRh)
Time frame: Up to 4 years and 9 months
Rate of CR/CRh is defined as the percentage of participants achieving a CR or CRh at any time post-treatment.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Age 2 years and above (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 less than or equal to (\<=) 20\*10\^9/liter (L) and (b) renal function; For adult participants, estimated or measured glomerular filtration rate greater
Where
- Duarte, California
- Orange, California
- San Francisco, California
- Chicago, Illinois
- Indianapolis, Indiana
- Louisville, Kentucky
- Boston, Massachusetts
- Detroit, Michigan
- Grand Rapids, Michigan
- Albuquerque, New Mexico
- Buffalo, New York
- New York, New York
And 11 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 6, 2026 · Source of record for eligibility and locations