RecruitingRecruiting
A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia (cAMeLot-1)
NCT04811560 · Janssen Research & Development, LLC
In plain English
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Official title
A Phase 1/2, First-in-Human Study of the Menin-KMT2A (MLL1) Inhibitor Bleximenib in Participants With Acute Leukemia (cAMeLot-1)
About this study
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
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
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
Enrollment target: 420 participants
Allocation: na
Masking: none
Age groups: child, adult, older_adult
Timeline
Starts: 2021-05-19
Estimated completion: 2030-09-22
Last updated: 2026-05-08
Interventions
Drug: Bleximenib
Primary outcomes
- • Phase 1: Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability (Up to 4 years and 9 months)
- • Phase 1: Number of Participants with AEs by Severity (Up to 4 years and 9 months)
- • Phase 1: Part 1: Percentage of Participants with Dose-Limiting Toxicity (DLT) (Up to 28 days Cycle 1)
Sponsor
Janssen Research & Development, LLC · industry
Contacts & investigators
ContactStudy Contact · contact · Participate-In-This-Study1@its.jnj.com · 844-434-4210
InvestigatorJanssen Research & Development, LLC Clinical Trial · study_director, Janssen Research & Development, LLC
All locations (103)
City of Hope PhoenixTerminated
Goodyear, Arizona, United States
City of HopeRecruiting
Duarte, California, United States
University of California Irvine Medical CenterCompleted
Orange, California, United States
University of California San FranciscoRecruiting
San Francisco, California, United States
UCSF Benioff Children's HospitalRecruiting
San Francisco, California, United States
University of ChicagoRecruiting
Chicago, Illinois, United States
St Francis Hospital & Health Centers Indiana Blood and Marrow TransplantationRecruiting
Indianapolis, Indiana, United States
Norton Cancer InstituteCompleted
Louisville, Kentucky, United States
Massachusetts General HospitalRecruiting
Boston, Massachusetts, United States
Dana Farber Cancer InstituteRecruiting
Boston, Massachusetts, United States
Karmanos Cancer InstituteRecruiting
Detroit, Michigan, United States
Start MidwestRecruiting
Grand Rapids, Michigan, United States
University of New MexicoRecruiting
Albuquerque, New Mexico, United States
Roswell Park Comprehensive Cancer CenterRecruiting
Buffalo, New York, United States
NYU Langone Medical CenterRecruiting
New York, New York, United States
Montefiore Medical CenterRecruiting
The Bronx, New York, United States
Oregon Health And Science UniversityRecruiting
Portland, Oregon, United States
Fox Chase Cancer CenterRecruiting
Philadelphia, Pennsylvania, United States
Maine HealthRecruiting
Providence, Rhode Island, United States
University of Tennessee Medical CenterRecruiting
Knoxville, Tennessee, United States
Baylor University Medical CenterRecruiting
Dallas, Texas, United States
Houston Methodist HospitalRecruiting
Houston, Texas, United States
MD AndersonRecruiting
Houston, Texas, United States
San Antonio Methodist TX Transplant Physicians GroupRecruiting
San Antonio, Texas, United States
Virginia Commonwealth UniversityRecruiting
Richmond, Virginia, United States
Swedish Cancer InstituteRecruiting
Seattle, Washington, United States
Medical College of WI at FroedtertRecruiting
Milwaukee, Wisconsin, United States
Monash Medical CentreRecruiting
Clayton, Australia
Royal Perth HospitalRecruiting
Perth, Australia
Gold Coast University HospitalRecruiting
Southport, Australia
Ghent University HospitalRecruiting
Ghent, Belgium
Instituto D Or de Pesquisa e EnsinoRecruiting
Brasília, Brazil
Liga Norte Riograndense Contra O CancerRecruiting
Natal, Brazil
Ministerio da Saude Instituto Nacional do CancerRecruiting
Rio de Janeiro, Brazil
Fundacao Faculdade de Medicina Instituto do Cancer do Estado de Sao PauloRecruiting
São Paulo, Brazil
Instituto D Or de Pesquisa e Ensino IDORRecruiting
São Paulo, Brazil
Fundacao Antonio Prudente A C Camargo Cancer CenterRecruiting
São Paulo, Brazil
Vancouver Coastal HealthRecruiting
Vancouver, British Columbia, Canada
Princess Margaret Cancer Centre University Health NetworkRecruiting
Toronto, Ontario, Canada
Hopital Maisonneuve RosemontRecruiting
Montreal, Quebec, Canada
Peking University First HospitalRecruiting
Beijing, China
The First Hospital of Jilin UniversityRecruiting
Changchun, China
West China Hospital Si Chuan UniversityRecruiting
Chengdu, China
Nanfang Hospital of Southern Medical HospitalRecruiting
Guangzhou, China
First Affiliated Hospital Medical School of Zhejiang UniversityRecruiting
Hangzhou, China
Qilu Hospital of Shandong UniversityRecruiting
Jinan, China
The First Affiliated Hospital of NanChang UniversityRecruiting
Nanchang, China
Zhongda Hospital Southeast UniversityRecruiting
Nanjing, China
Institute of Hematology and Blood Diseases HospitalRecruiting
Tianjin, China
The First Affiliated Hospital of Wenzhou Medical UniversityRecruiting
Wenzhou, China
Union Hospital Tongji Medical College of Huazhong University of Science and TechnologyRecruiting
Wuhan, China
Henan Cancer HospitalRecruiting
Zhengzhou, China
Hopital Jean MinjozRecruiting
Besançon, France
Institut Paoli CalmettesRecruiting
Marseille, France
CHU de Nantes hotel DieuRecruiting
Nantes, France
Hopital Saint LouisRecruiting
Paris, France
Hopital trousseau- APHPRecruiting
Paris, France
Centre Hospitalier Universitaire (CHU) de Bordeaux Hopital HautLeveque Centre Francois MagendieCompleted
Pessac, France
CHU Lyon SudRecruiting
Pierre-Bénite, France
Hôpitaux Universitaires de Strasbourg - Hôpital de HautepierreRecruiting
Strasbourg, France
Institut Universitaire du Cancer Toulouse OncopoleRecruiting
Toulouse, France
CHU BretonneauRecruiting
Tours, France
CHRU de Nancy - Hopitaux de BraboisRecruiting
Vandœuvre-lès-Nancy, France
CHRU Nancy BraboisRecruiting
Vandœuvre-lès-Nancy, France
Gustave RoussyRecruiting
Villejuif, France
Carmel Medical CenterRecruiting
Haifa, Israel
Hadassah University Hospita Ein KeremRecruiting
Jerusalem, Israel
Sheba Medical CenterRecruiting
Ramat Gan, Israel
Tel Aviv Sourasky Medical CenterRecruiting
Tel Aviv, Israel
Kyushu University HospitalActive Not Recruiting
Fukuoka, Japan
Fukushima Medical University HospitalActive Not Recruiting
Fukushima, Japan
Kanazawa University HospitalActive Not Recruiting
Kanazawa, Japan
National Cancer Center Hospital EastActive Not Recruiting
Kashiwa, Japan
Kobe City Medical Center General HospitalActive Not Recruiting
Kobe, Japan
Gunmaken Saiseikai Maebashi HospitalActive Not Recruiting
Maebashi, Japan
Nagoya University HospitalCompleted
Nagoya, Japan
Hokkaido University HospitalActive Not Recruiting
Sapporo, Japan
NTT Medical Center TokyoActive Not Recruiting
Tokyo, Japan
Yamagata University HospitalCompleted
Yamagata, Japan
University of Fukui HospitalCompleted
Yoshida, Japan
Seoul National University HospitalRecruiting
Seoul, South Korea
Asan Medical CenterRecruiting
Seoul, South Korea
Samsung Medical CenterRecruiting
Seoul, South Korea
Hosp Univ Vall D HebronRecruiting
Barcelona, Spain
Hosp Clinic de BarcelonaRecruiting
Barcelona, Spain
Hosp. Gral. Univ. Gregorio MaranonRecruiting
Madrid, Spain
Hospital Infantil Universitario Nino JesusRecruiting
Madrid, Spain
Hosp Univ Fund Jimenez DiazRecruiting
Madrid, Spain
Clinica Univ. de NavarraRecruiting
Pamplona, Spain
Hosp Clinico Univ de SalamancaRecruiting
Salamanca, Spain
Hosp. Virgen Del RocioRecruiting
Seville, Spain
Hosp. Clinico Univ. de ValenciaRecruiting
Valencia, Spain
China Medical University HospitalRecruiting
Taichung, Taiwan
National Cheng Kung University HospitalRecruiting
Tainan, Taiwan
National Taiwan University HospitalRecruiting
Taipei, Taiwan
University Hospital of WalesRecruiting
Cardiff, United Kingdom
Leeds Teaching Hospitals NHS TrustRecruiting
Leeds, United Kingdom
The Clatterbridge Cancer CentreRecruiting
Liverpool, United Kingdom
Guys and St Thomas NHS Foundation TrustRecruiting
London, United Kingdom
University College London HospitalsRecruiting
London, United Kingdom
The Christie Nhs Foundation TrustRecruiting
Manchester, United Kingdom
Oxford University Hospitals NHS TrustRecruiting
Oxfordshire, United Kingdom
University Hospitals Plymouth NHS TrustRecruiting
Plymouth, United Kingdom