Clinical Trials List
Protocol Number75276617ALE1001
NCT Number(ClinicalTrials.gov Identfier)NCT04811560
Active
2024-09-01 - 2028-03-01
Phase II
Not yet recruiting1
Recruiting2
ICD-10C95.00
Acute leukemia of unspecified cell type not having achieved remission
ICD-10C95.02
Acute leukemia of unspecified cell type, in relapse
ICD-10Z51.12
Encounter for antineoplastic immunotherapy
ICD-9208.00
Acute leukemia of unspecified cell type, without mention of remission
A Phase 1/2, First-in-Human Study of the Menin-KMT2A (MLL1) Inhibitor Bleximenib in Participants With Acute Leukemia (cAMeLot-1)
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Trial Applicant
Johnson & Johnson
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Sponsor
-
Trial scale
Multi-Regional Multi-Center
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Update
2026/06/17
Investigators and Locations
Co-Principal Investigator
- Wen-Chien Chou Division of Hematology & Oncology
- MING YAO Division of Hematology & Oncology
- CHENG-HONG TSAI Division of General Internal Medicine
- 李思慧 Division of Hematology & Oncology
- Huai-Hsuan Huang Division of Hematology & Oncology
- 林明恩 Division of General Internal Medicine
- YAO CHI-YUAN Division of General Internal Medicine
- Chieh-Lung Cheng Division of General Internal Medicine
- 田豐銘 Division of General Internal Medicine
- Chien-Chin Lin Division of Others -
- - - Division of Hematology & Oncology
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
- Che-Hung Lin Division of Hematology & Oncology
- 王幸婷 Division of Hematology & Oncology
- 陳珈妤 Division of Hematology & Oncology
- Ming-Yu Lien Division of Hematology & Oncology
- Yu-Min Liao Division of Hematology & Oncology
- 王秀慈 Division of Hematology & Oncology
- Chi-Ching Chen Division of Hematology & Oncology
- 鄭富銘 Division of Hematology & Oncology
The Actual Total Number of Participants Enrolled
0 Recruiting
Principal Investigator
Tsai-Yun Chen
Co-Principal Investigator
- Sin-Syue Li Division of Hematology & Oncology
- 傅蓓安 Division of Hematology & Oncology
- Ya-Ting Hsu Division of Hematology & Oncology
- Ya-Ping Chen Division of Hematology & Oncology
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Acute Leukemia
Objectives
The primary objective of Phase 1 was to determine the RP2D of bleximenib and its safety and tolerability. The primary objective of Phase 2 was to evaluate the efficacy of bleximenib under RP2D. (Taiwan only participated in Phase 2 trials.)
Test Drug
Tablets
Active Ingredient
Bleximenib
Dosage Form
110
Dosage
001
Endpoints
Complete remission or complete remission with partial recovery of blood cell count (CR/CRh) rate: up to 4 years and 9 months; CR/CRh rate will be assessed and reported up to 4 years and 9 months based on the evaluation of an Independent Review Committee (IRC). CR/CRh rate is defined as the percentage of subjects who achieve complete remission (CR) or complete remission with partial recovery of blood cell count (CRh) at any time after treatment.
Inclution Criteria
Inclusion Criteria:
Phase 1:
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
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 <= 20*10^9/liter (L) and (b) renal function; For adult participants, estimated or measured glomerular filtration rate >= 30 milliliter per minute (mL/min) per four variable MDRD equation. For pediatric participants an estimated or measured glomerular filtration rate >=40 mL/min per the CKiD (Chronic Kidney Disease in Children) Schwartz formula
Eastern Cooperative Oncology Group (ECOG) performance status grade of 0, 1 or 2. Pediatric participants only: Performance status >=70 by Lansky scale (for participants < 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
Phase 1:
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
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 <= 20*10^9/liter (L) and (b) renal function; For adult participants, estimated or measured glomerular filtration rate >= 30 milliliter per minute (mL/min) per four variable MDRD equation. For pediatric participants an estimated or measured glomerular filtration rate >=40 mL/min per the CKiD (Chronic Kidney Disease in Children) Schwartz formula
Eastern Cooperative Oncology Group (ECOG) performance status grade of 0, 1 or 2. Pediatric participants only: Performance status >=70 by Lansky scale (for participants < 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
Exclusion Criteria
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)
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)
The Estimated Number of Participants
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Taiwan
15 participants
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Global
200 participants
