Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
MY TRIALS
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Register a trial
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04811560
Registration number
NCT04811560
Ethics application status
Date submitted
22/03/2021
Date registered
23/03/2021
Titles & IDs
Public title
A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia
Query!
Scientific title
A Phase 1/2, First-in-Human Study of the Menin-KMT2A (MLL1) Inhibitor Bleximenib in Participants With Acute Leukemia
Query!
Secondary ID [1]
0
0
2020-005967-30
Query!
Secondary ID [2]
0
0
CR108998
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Acute Leukemias
0
0
Query!
Acute Myeloid Leukemia
0
0
Query!
Acute Lymphoblastic Leukemia
0
0
Query!
Condition category
Condition code
Cancer
0
0
0
0
Query!
Leukaemia - Acute leukaemia
Query!
Cancer
0
0
0
0
Query!
Leukaemia - Chronic leukaemia
Query!
Cancer
0
0
0
0
Query!
Children's - Leukaemia & Lymphoma
Query!
Cancer
0
0
0
0
Query!
Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma
Query!
Cancer
0
0
0
0
Query!
Lymphoma (non Hodgkin's lymphoma) - Low grade lymphoma
Query!
Other
0
0
0
0
Query!
Research that is not of generic health relevance and not applicable to specific health categories listed above
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - Bleximenib
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. Food effect cohort (optional) participants will receive bleximenib orally on Cycle 2 Day 1 under fasted condition and on Cycle 2 Day 2 under fed condition. Participants in Phase 2 part of the study will receive bleximenib orally at the RP2D level(s).
Treatment: Drugs: Bleximenib
Bleximenib is administered orally.
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Phase 1: Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Up to 4 years and 9 months
Query!
Primary outcome [2]
0
0
Phase 1: Number of Participants with AEs by Severity
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
Up to 4 years and 9 months
Query!
Primary outcome [3]
0
0
Phase 1: Part 1: Percentage of Participants with Dose-Limiting Toxicity (DLT)
Query!
Assessment method [3]
0
0
Percentage of participants with DLT will be assessed accordingly to national cancer institute common terminology criteria for adverse events (NCI CTCAE) version 5.
Query!
Timepoint [3]
0
0
Up to 28 days Cycle 1
Query!
Primary outcome [4]
0
0
Phase 2: Rate of Complete Remission or Complete Remission with Partial Hematologic Recovery (CR/CRh)
Query!
Assessment method [4]
0
0
Rate of CR/CRh is defined as the percentage of participants achieving a CR or CRh at any time post-treatment.
Query!
Timepoint [4]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [1]
0
0
Phase 1: Plasma Concentration of Bleximenib
Query!
Assessment method [1]
0
0
Plasma concentration of bleximenib will be reported.
Query!
Timepoint [1]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [2]
0
0
Phase 1 and 2: Overall Response Rate (ORR)
Query!
Assessment method [2]
0
0
ORR is based on investigator-determined responses and is defined as the percentage of participants who achieve any response.
Query!
Timepoint [2]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [3]
0
0
Phase 1 and 2: Duration of Response (DOR)
Query!
Assessment method [3]
0
0
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.
Query!
Timepoint [3]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [4]
0
0
Phase 1 and 2: Time To Response (TTR)
Query!
Assessment method [4]
0
0
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.
Query!
Timepoint [4]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [5]
0
0
Phase 2: Event-free survival (EFS)
Query!
Assessment method [5]
0
0
EFS is defined as the time from the date of first dose of study treatment to the date of treatment failure, relapse, or death due to any cause, whichever occurs first.
Query!
Timepoint [5]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [6]
0
0
Phase 2: Overall survival (OS)
Query!
Assessment method [6]
0
0
OS is defined from the date of first dose of study treatment to the date of death due to any cause.
Query!
Timepoint [6]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [7]
0
0
Phase 2: Measurable Residual Disease (MRD) Negativity Among Participants Achieving CR/CRh/CRi
Query!
Assessment method [7]
0
0
MRD-negative rate is defined as the percentage of participants who are MRD-negative at any timepoint after the first dose of bleximenib in the responders.
Query!
Timepoint [7]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [8]
0
0
Phase 2: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Query!
Assessment method [8]
0
0
An AE is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. A Serious AE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.
Query!
Timepoint [8]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [9]
0
0
Phase 2: Number of Participants Reporting Transfusion Independence
Query!
Assessment method [9]
0
0
Transfusion independence is defined as independence from red blood cells (RBC) and platelet transfusions during any 56-day interval after receiving study treatment.
Query!
Timepoint [9]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [10]
0
0
Phase 2: Maximum Plasma Concentration (Cmax) of Bleximenib
Query!
Assessment method [10]
0
0
Cmax is defined as maximum plasma concentration.
Query!
Timepoint [10]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [11]
0
0
Phase 2: Time to Reach the Maximum Concentration (Tmax) of Bleximenib
Query!
Assessment method [11]
0
0
Tmax is defined as time to reach the maximum concentration.
Query!
Timepoint [11]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [12]
0
0
Phase 2: Area Under the Plasma Concentration-time Curve (AUC) from Time T1 to T2 of Bleximenib
Query!
Assessment method [12]
0
0
AUC t1-t2 is defined as area under the plasma concentration-time curve from time t1 to t2.
Query!
Timepoint [12]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [13]
0
0
Phase 2: Area Under The Curve Over a Dosing Interval (AUCtau) of Bleximenib
Query!
Assessment method [13]
0
0
AUCtau is defined as area under the curve over a dosing interval.
Query!
Timepoint [13]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [14]
0
0
Phase 2: Apparent Clearance (CL/F) of Bleximenib
Query!
Assessment method [14]
0
0
CL/F is defined as a measure of the rate at which a drug got metabolized or eliminated by normal biological processes.
Query!
Timepoint [14]
0
0
Up to 4 years and 9 months
Query!
Secondary outcome [15]
0
0
Phase 2: Apparent Volume of Distribution (V/F) of Bleximenib
Query!
Assessment method [15]
0
0
V/F is defined as a theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired serum concentration of a drug.
Query!
Timepoint [15]
0
0
Up to 4 years and 9 months
Query!
Eligibility
Key inclusion criteria
Phase 1:
* Relapsed or refractory (R/R) acute leukemia and has exhausted, or is ineligible for, available therapeutic options
* Participants greater than or equal (>=)12 and less than (<) 18 years of age with a body weight of >= 40 kg are only eligible for the Phase 1 adolescent cohort
* 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.
* 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 renal function; Estimated or measured glomerular filtration rate greater than or equal to (>=) 50 milliliter per minute (mL/min) per four variable modified diet in renal disease (MDRD) equation
* Eastern Cooperative Oncology Group (ECOG) performance status grade of 0, 1 or 2. Adolescent participants only: Performance status >=70 by Lansky scale (for participants less than [<]16 years of age) or >=70 Karnofsky scale (for participants >=16 years of age)
* A participant 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
* A participant must agree to all the following during the study and for 90 days after the last dose of study treatment: (a) wear a condom when engaging in any activity that allows for passage of ejaculate to another person; (b) not to donate sperm or freeze for future use for the purpose of reproduction. In addition, the participant should be advised of the benefit for a partner to use a highly effective method of contraception as condom may break or leak
Query!
Minimum age
12
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key 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)
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
NA
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Other
Query!
Other design features
Query!
Phase
Phase 1
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Recruiting
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
19/05/2021
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
30/10/2027
Query!
Actual
Query!
Sample size
Target
350
Query!
Accrual to date
Query!
Final
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment hospital [1]
0
0
Monash Medical Centre - Clayton
Query!
Recruitment hospital [2]
0
0
Royal Perth Hospital - Perth
Query!
Recruitment hospital [3]
0
0
Gold Coast University Hospital - Southport
Query!
Recruitment postcode(s) [1]
0
0
3168 - Clayton
Query!
Recruitment postcode(s) [2]
0
0
6000 - Perth
Query!
Recruitment postcode(s) [3]
0
0
4211 - Southport
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Kentucky
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Massachusetts
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Michigan
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
New York
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Texas
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Wisconsin
Query!
Country [8]
0
0
France
Query!
State/province [8]
0
0
Marseille
Query!
Country [9]
0
0
France
Query!
State/province [9]
0
0
Nantes Cedex 1
Query!
Country [10]
0
0
France
Query!
State/province [10]
0
0
Paris
Query!
Country [11]
0
0
France
Query!
State/province [11]
0
0
Pessac
Query!
Country [12]
0
0
France
Query!
State/province [12]
0
0
Toulouse
Query!
Country [13]
0
0
France
Query!
State/province [13]
0
0
Tours cedex
Query!
Country [14]
0
0
Spain
Query!
State/province [14]
0
0
Barcelona
Query!
Country [15]
0
0
Spain
Query!
State/province [15]
0
0
Madrid
Query!
Country [16]
0
0
Spain
Query!
State/province [16]
0
0
Pamplona
Query!
Country [17]
0
0
United Kingdom
Query!
State/province [17]
0
0
London
Query!
Country [18]
0
0
United Kingdom
Query!
State/province [18]
0
0
Manchester
Query!
Country [19]
0
0
United Kingdom
Query!
State/province [19]
0
0
Oxfordshire
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Janssen Research & Development, LLC
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
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.
Query!
Trial website
https://clinicaltrials.gov/study/NCT04811560
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Janssen Research & Development, LLC Clinical Trial
Query!
Address
0
0
Janssen Research & Development, LLC
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Study Contact
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
844-434-4210
Query!
Fax
0
0
Query!
Email
0
0
[email protected]
Query!
Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
Query!
What data in particular will be shared?
The data sharing policy of the Janssen Pharmaceutical Companies of Johnson \& Johnson is available at www.janssen.com/clinical-trials/transparency.
As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu
Query!
When will data be available (start and end dates)?
Query!
Available to whom?
Query!
Available for what types of analyses?
Query!
How or where can data be obtained?
IPD available at link: https://www.janssen.com/clinical-trials/transparency
Query!
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT04811560