A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia (cAMeLot-1)
ConditionAcute Leukemia
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age2+
SponsorJanssen Research & Development, LLC
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Participants greater than 18 years are eligible
Disqualifiers
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
Trial design
Design model
Sequential
Treatments tested in this trial
Bleximenib
DrugBleximenib is administered orally.
Treatment groups
420 Participants
are divided into 1 treatment groupGroup A: BleximenibExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Phase 1: Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability
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.
Time frame
Up to 4 years and 9 months2
Phase 1: Number of Participants with AEs by Severity
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.
Time frame
Up to 4 years and 9 months3
Phase 1: Part 1: Percentage of Participants with Dose-Limiting Toxicity (DLT)
Percentage of participants with DLT will be assessed accordingly to national cancer institute common terminology criteria for adverse events (NCI-CTCAE) version 5.
Time frame
Up to 28 days Cycle 14
Phase 2: Rate of Complete Remission or Complete Remission with Partial Hematologic Recovery (CR/CRh)
Rate of CR/CRh is defined as the percentage of participants achieving a CR or CRh at any time post-treatment.
Time frame
Up to 4 years and 9 monthsSecondary outcomes
1
Phase 1 and 2: Plasma Concentration of Bleximenib
Plasma concentration of bleximenib will be reported.
Time frame
Up to 4 years and 9 months2
Phase 1 and 2: Overall Response Rate (ORR)
ORR is defined as the percentage of participants who achieve any response.
Time frame
Up to 4 years and 9 months3
Phase 1: Duration of Response (DOR)
DOR will be calculated among responders from the date of initial documentation of a response to the date of first documented evidence of relapse, as defined in the disease-specific response criteria, or death due to any cause, whichever occurs first.
Time frame
Up to 4 years and 9 months4
Phase 1 and 2: Time To Response (TTR)
TTR is defined for the responders as the time from the date of the first dose of bleximenib to the date of the first documented response.
Time frame
Up to 4 years and 9 monthsSponsors and contacts
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Source ClinicalTrials.gov