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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
Date last updated
22/06/2025
Titles & IDs
Public title
A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia
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Scientific title
A Phase 1/2, First-in-Human Study of the Menin-KMT2A (MLL1) Inhibitor Bleximenib in Participants With Acute Leukemia
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Secondary ID [1]
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75276617ALE1001
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Secondary ID [2]
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CR108998
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Acute Leukemias
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Acute Myeloid Leukemia
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Acute Lymphoblastic Leukemia
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Condition category
Condition code
Cancer
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Leukaemia - Acute leukaemia
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Cancer
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Leukaemia - Chronic leukaemia
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Cancer
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Children's - Leukaemia & Lymphoma
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Cancer
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Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma
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Cancer
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Lymphoma (non Hodgkin's lymphoma) - Low grade lymphoma
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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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.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Phase 1: Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability
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Assessment method [1]
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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.
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Timepoint [1]
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Up to 4 years and 9 months
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Primary outcome [2]
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Phase 1: Number of Participants with AEs by Severity
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Assessment method [2]
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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.
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Timepoint [2]
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Up to 4 years and 9 months
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Primary outcome [3]
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Phase 1: Part 1: Percentage of Participants with Dose-Limiting Toxicity (DLT)
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Assessment method [3]
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Percentage of participants with DLT will be assessed accordingly to national cancer institute common terminology criteria for adverse events (NCI CTCAE) version 5.
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Timepoint [3]
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Up to 28 days Cycle 1
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Primary outcome [4]
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Phase 2: Rate of Complete Remission or Complete Remission with Partial Hematologic Recovery (CR/CRh)
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Assessment method [4]
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Rate of CR/CRh is defined as the percentage of participants achieving a CR or CRh at any time post-treatment.
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Timepoint [4]
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Up to 4 years and 9 months
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Secondary outcome [1]
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Phase 1 and 2: Plasma Concentration of Bleximenib
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Assessment method [1]
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Plasma concentration of bleximenib will be reported.
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Timepoint [1]
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Up to 4 years and 9 months
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Secondary outcome [2]
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Phase 1 and 2: Overall Response Rate (ORR)
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Assessment method [2]
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ORR is defined as the percentage of participants who achieve any response.
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Timepoint [2]
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Up to 4 years and 9 months
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Secondary outcome [3]
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Phase 1 and 2: Duration of Response (DOR)
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Assessment method [3]
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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.
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Timepoint [3]
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Up to 4 years and 9 months
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Secondary outcome [4]
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Phase 1 and 2: Time To Response (TTR)
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Assessment method [4]
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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.
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Timepoint [4]
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Up to 4 years and 9 months
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Secondary outcome [5]
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Phase 2: Duration of Complete Response (CR)/Complete Remission With Partial Hematologic Recovery (CRh)
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Assessment method [5]
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The duration of CR/CRh is defined from the date of first CR or CRh response achieved to the date of first evidence of relapsed disease or death due to any cause, whichever occurs first, for participants who achieve a CR or CRh.
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Timepoint [5]
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Up to 4 years and 9 months
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Secondary outcome [6]
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Phase 2: Time To CR/CRh
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Assessment method [6]
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Time to CR/CRh is defined for responders as the time from the date of the first dose of bleximenib to the date of first achieving either CR or CRh, depending on which milestone is reached.
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Timepoint [6]
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Up to 4 years and 9 months
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Secondary outcome [7]
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Phase 2: Event-free survival (EFS)
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Assessment method [7]
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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.
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Timepoint [7]
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Up to 4 years and 9 months
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Secondary outcome [8]
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Phase 2: Overall survival (OS)
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Assessment method [8]
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OS is defined from the date of first dose of study treatment to the date of death due to any cause.
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Timepoint [8]
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Up to 4 years and 9 months
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Secondary outcome [9]
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Phase 2: Measurable Residual Disease (MRD) Negativity Among Participants Achieving CR/CRh/CRi
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Assessment method [9]
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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.
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Timepoint [9]
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Up to 4 years and 9 months
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Secondary outcome [10]
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Phase 2: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
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Assessment method [10]
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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.
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Timepoint [10]
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Up to 4 years and 9 months
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Secondary outcome [11]
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Phase 2: Number of Participants Reporting Transfusion Independence
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Assessment method [11]
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Transfusion independence is defined as independence from red blood cells (RBC) and platelet transfusions during any 56-day interval after receiving study treatment.
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Timepoint [11]
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Up to 4 years and 9 months
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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
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Minimum age
12
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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)
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Not applicable
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Other
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
19/05/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
26/05/2028
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Actual
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Sample size
Target
400
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Monash Medical Centre - Clayton
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Recruitment hospital [2]
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Royal Perth Hospital - Perth
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Recruitment hospital [3]
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Gold Coast University Hospital - Southport
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Recruitment postcode(s) [1]
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3168 - Clayton
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Recruitment postcode(s) [2]
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6000 - Perth
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Recruitment postcode(s) [3]
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4211 - Southport
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Recruitment outside Australia
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United States of America
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California
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Illinois
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United States of America
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Indiana
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Kentucky
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United States of America
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Massachusetts
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Michigan
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New Mexico
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New York
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Oregon
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Pennsylvania
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Tennessee
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Texas
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Brasilia
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Brazil
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Natal
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Brazil
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Rio de Janeiro
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Brazil
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Canada
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China
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Hang Zhou
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China
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Jinan
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Nanjing
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China
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Tian Jin
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China
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China
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Wuhan
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China
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Zheng Zhou Shi
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France
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Besancon
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Marseille
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Pessac
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France
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Pierre Benite
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France
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Israel
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Israel
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Ramat Gan
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Israel
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Fukuoka
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Tokyo
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Yamagata
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Yoshida
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Korea, Republic of
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Seoul
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Valencia
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Taichung
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Taipei
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Cardiff
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Manchester
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Oxfordshire
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United Kingdom
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Plymouth
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Janssen Research & Development, LLC
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Address
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Country
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Ethics approval
Ethics application status
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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.
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Trial website
https://clinicaltrials.gov/study/NCT04811560
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Janssen Research & Development, LLC Clinical Trial
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Address
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Janssen Research & Development, LLC
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Study Contact
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Address
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Phone
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844-434-4210
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Fax
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Email
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[email protected]
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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
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