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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
Scientific title
A Phase 1/2, First-in-Human Study of the Menin-KMT2A (MLL1) Inhibitor Bleximenib in Participants With Acute Leukemia
Secondary ID [1] 0 0
75276617ALE1001
Secondary ID [2] 0 0
CR108998
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Acute Leukemias 0 0
Acute Myeloid Leukemia 0 0
Acute Lymphoblastic Leukemia 0 0
Condition category
Condition code
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma
Cancer 0 0 0 0
Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma
Cancer 0 0 0 0
Lymphoma (non Hodgkin's lymphoma) - Low grade lymphoma
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
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.


Treatment: Drugs: Bleximenib
Bleximenib is administered orally.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Phase 1: Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability
Timepoint [1] 0 0
Up to 4 years and 9 months
Primary outcome [2] 0 0
Phase 1: Number of Participants with AEs by Severity
Timepoint [2] 0 0
Up to 4 years and 9 months
Primary outcome [3] 0 0
Phase 1: Part 1: Percentage of Participants with Dose-Limiting Toxicity (DLT)
Timepoint [3] 0 0
Up to 28 days Cycle 1
Primary outcome [4] 0 0
Phase 2: Rate of Complete Remission or Complete Remission with Partial Hematologic Recovery (CR/CRh)
Timepoint [4] 0 0
Up to 4 years and 9 months
Secondary outcome [1] 0 0
Phase 1 and 2: Plasma Concentration of Bleximenib
Timepoint [1] 0 0
Up to 4 years and 9 months
Secondary outcome [2] 0 0
Phase 1 and 2: Overall Response Rate (ORR)
Timepoint [2] 0 0
Up to 4 years and 9 months
Secondary outcome [3] 0 0
Phase 1 and 2: Duration of Response (DOR)
Timepoint [3] 0 0
Up to 4 years and 9 months
Secondary outcome [4] 0 0
Phase 1 and 2: Time To Response (TTR)
Timepoint [4] 0 0
Up to 4 years and 9 months
Secondary outcome [5] 0 0
Phase 2: Duration of Complete Response (CR)/Complete Remission With Partial Hematologic Recovery (CRh)
Timepoint [5] 0 0
Up to 4 years and 9 months
Secondary outcome [6] 0 0
Phase 2: Time To CR/CRh
Timepoint [6] 0 0
Up to 4 years and 9 months
Secondary outcome [7] 0 0
Phase 2: Event-free survival (EFS)
Timepoint [7] 0 0
Up to 4 years and 9 months
Secondary outcome [8] 0 0
Phase 2: Overall survival (OS)
Timepoint [8] 0 0
Up to 4 years and 9 months
Secondary outcome [9] 0 0
Phase 2: Measurable Residual Disease (MRD) Negativity Among Participants Achieving CR/CRh/CRi
Timepoint [9] 0 0
Up to 4 years and 9 months
Secondary outcome [10] 0 0
Phase 2: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [10] 0 0
Up to 4 years and 9 months
Secondary outcome [11] 0 0
Phase 2: Number of Participants Reporting Transfusion Independence
Timepoint [11] 0 0
Up to 4 years and 9 months

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 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 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; 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 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
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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)

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
19/05/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/10/2027
Actual
Sample size
Target
400
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Monash Medical Centre - Clayton
Recruitment hospital [2] 0 0
Royal Perth Hospital - Perth
Recruitment hospital [3] 0 0
Gold Coast University Hospital - Southport
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment postcode(s) [2] 0 0
6000 - Perth
Recruitment postcode(s) [3] 0 0
4211 - Southport
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
Indiana
Country [4] 0 0
United States of America
State/province [4] 0 0
Kentucky
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
New Mexico
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
Oregon
Country [10] 0 0
United States of America
State/province [10] 0 0
Pennsylvania
Country [11] 0 0
United States of America
State/province [11] 0 0
Rhode Island
Country [12] 0 0
United States of America
State/province [12] 0 0
Tennessee
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
United States of America
State/province [14] 0 0
Virginia
Country [15] 0 0
United States of America
State/province [15] 0 0
Washington
Country [16] 0 0
United States of America
State/province [16] 0 0
Wisconsin
Country [17] 0 0
Brazil
State/province [17] 0 0
Sao Paulo
Country [18] 0 0
Canada
State/province [18] 0 0
British Columbia
Country [19] 0 0
Canada
State/province [19] 0 0
Ontario
Country [20] 0 0
Canada
State/province [20] 0 0
Quebec
Country [21] 0 0
China
State/province [21] 0 0
Beijing
Country [22] 0 0
China
State/province [22] 0 0
Changchun
Country [23] 0 0
China
State/province [23] 0 0
Chengdu
Country [24] 0 0
China
State/province [24] 0 0
Guangzhou
Country [25] 0 0
China
State/province [25] 0 0
Hang Zhou
Country [26] 0 0
China
State/province [26] 0 0
Jinan
Country [27] 0 0
China
State/province [27] 0 0
Nanchang
Country [28] 0 0
China
State/province [28] 0 0
Nanjing
Country [29] 0 0
China
State/province [29] 0 0
Tian Jin
Country [30] 0 0
China
State/province [30] 0 0
Wenzhou
Country [31] 0 0
China
State/province [31] 0 0
Wuhan
Country [32] 0 0
China
State/province [32] 0 0
Zheng Zhou Shi
Country [33] 0 0
France
State/province [33] 0 0
Besancon
Country [34] 0 0
France
State/province [34] 0 0
Marseille
Country [35] 0 0
France
State/province [35] 0 0
Nantes Cedex 1
Country [36] 0 0
France
State/province [36] 0 0
Paris
Country [37] 0 0
France
State/province [37] 0 0
Pessac
Country [38] 0 0
France
State/province [38] 0 0
Pierre Benite
Country [39] 0 0
France
State/province [39] 0 0
Strasbourg
Country [40] 0 0
France
State/province [40] 0 0
Toulouse
Country [41] 0 0
France
State/province [41] 0 0
Tours cedex
Country [42] 0 0
France
State/province [42] 0 0
Vandoeuvre les Nancy
Country [43] 0 0
France
State/province [43] 0 0
Villejuif
Country [44] 0 0
Israel
State/province [44] 0 0
Haifa
Country [45] 0 0
Israel
State/province [45] 0 0
Jerusalem
Country [46] 0 0
Israel
State/province [46] 0 0
Ramat Gan
Country [47] 0 0
Israel
State/province [47] 0 0
Tel Aviv Yafo
Country [48] 0 0
Japan
State/province [48] 0 0
Fukushima
Country [49] 0 0
Japan
State/province [49] 0 0
Nagoya
Country [50] 0 0
Japan
State/province [50] 0 0
Sapporo
Country [51] 0 0
Japan
State/province [51] 0 0
Tokyo
Country [52] 0 0
Japan
State/province [52] 0 0
Yoshida
Country [53] 0 0
Korea, Republic of
State/province [53] 0 0
Seoul
Country [54] 0 0
Spain
State/province [54] 0 0
Barcelona
Country [55] 0 0
Spain
State/province [55] 0 0
Madrid
Country [56] 0 0
Spain
State/province [56] 0 0
Pamplona
Country [57] 0 0
Spain
State/province [57] 0 0
Salamanca
Country [58] 0 0
Spain
State/province [58] 0 0
Sevilla
Country [59] 0 0
Spain
State/province [59] 0 0
Valencia
Country [60] 0 0
Taiwan
State/province [60] 0 0
Taichung
Country [61] 0 0
Taiwan
State/province [61] 0 0
Tainan
Country [62] 0 0
Taiwan
State/province [62] 0 0
Taipei
Country [63] 0 0
United Kingdom
State/province [63] 0 0
London
Country [64] 0 0
United Kingdom
State/province [64] 0 0
Manchester
Country [65] 0 0
United Kingdom
State/province [65] 0 0
Oxfordshire
Country [66] 0 0
United Kingdom
State/province [66] 0 0
Plymouth

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Janssen Research & Development, LLC
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Janssen Research & Development, LLC Clinical Trial
Address 0 0
Janssen Research & Development, LLC
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Study Contact
Address 0 0
Country 0 0
Phone 0 0
844-434-4210
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


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