Clinical trial • Phase I • Oncology

BLEXIMENIB for Acute Myeloid Leukemia

Phase I trial of BLEXIMENIB for Acute Myeloid Leukemia. adaptive. 60 participants.

Overview

Trial Therapeutic Area
Oncology
Trial Disease
Acute Myeloid Leukemia
Trial Stage
Phase I
Drug Modality
Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
01-02-2024
First CTIS Authorization Date
18-03-2024

Trial design

adaptive Phase I trial across 22 sites in France, Germany, Italy and others.

Adaptive
True: Dose-finding/dose-escalation design to determine recommended RP2D candidate(s) followed by dose-expansion cohorts to further evaluate safety and tolerability at the RP2D(s).
Biomarker Stratified
True: KMT2A, NPM1, NUP98, NUP214
Single Multiple Or Escalation Dose Combined
Yes
Target Sample Size
60

Eligibility

Recruits 60 paediatric patients.

Pregnancy Exclusion
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
Vulnerable Population
Vulnerable population selected. Informed consent required: "Must sign an informed consent form (ICF) indicating participant (or their LAR) understands the purpose of the study and procedures required for the study and is willing to participate in the study." Consent may be provided by the participant or their legally authorised representative (LAR). Paediatric sites are included (paediatric oncology departments listed), indicating additional vulnerable-subject considerations.

Inclusion criteria

  • {"criterion_text":"- Diagnosis of AML according to World Health Organization (WHO) criteria: a) De novo or secondary AML; b) relapsed /refractory (Arm A only); c) harboring NPM1, KMT2A, NUP98, or NUP214 alterations"}
  • {"criterion_text":"- Pretreatment clinical laboratory values meeting the following criteria -listed below: White blood cell (WBC) count: less than or equal to <=25 x 10^9 per liter (/L), adequate liver and renal function"}
  • {"criterion_text":"- ECOG performance status grade of 0, 1 or 2"}
  • {"criterion_text":"- 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"}
  • {"criterion_text":"- Must sign an informed consent form (ICF) indicating participant (or their LAR) understands the purpose of the study and procedures required for the study and is willing to participate in the study."}
  • {"criterion_text":"- Willing and able to adhere to the prohibitions and restrictions specified in this protocol"}

Exclusion criteria

  • {"criterion_text":"- 1. Acute promyelocytic leukemia diagnosis of Down syndrome associated leukemia or juvenile myelomonocytic leukemia according to WHO 2016 criteria"}
  • {"criterion_text":"- 2. Leukemic involvement of the central nervous system"}
  • {"criterion_text":"- 3. Recipient of solid organ transplant"}
  • {"criterion_text":"- 5. Cardiovascular disease that is uncontrolled, increases risk for Torsades de Pointes or that was diagnosed within 6 months prior to the first dose of study treatment"}
  • {"criterion_text":"- Any toxicity (except for alopecia, stable peripheral neuropathy, thrombocytopenia, neutropenia, anemia) from previous anticancer therapy that has not resolved to baseline or to grade 1 or less."}
  • {"criterion_text":"- Pulmonary compromise that requires the need for supplemental oxygen use to maintain adequate oxygenation"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- 1. Incidence and severity of AEs, including DLTs","definition_or_measurement_approach":""}
  • {"endpoint_text":"- 2. Incidence and severity of Aes","definition_or_measurement_approach":""}

Recruitment

Planned Sample Size
86
Recruitment Window Months
46
Consent Approach
Must sign an informed consent form (ICF) indicating participant (or their LAR) understands the purpose of the study and procedures required for the study and is willing to participate in the study.

Geography

Total Number Of Sites
22
Total Number Of Participants
86

France

Earliest CTIS Part Ii Submission Date
04-01-2024
Latest Decision Or Authorization Date
30-09-2025
Processing Time Days
636
Number Of Sites
5
Number Of Participants
18

Sites

Site Name
Institut Universitaire Du Cancer Toulouse-Oncopole
Department Name
Hematology
Principal Investigator Name
Christian Recher
Principal Investigator Email
Recher.Christian@iuct-oncopole.fr
Contact Person Name
Christian Recher
Site Name
Centre Hospitalier Regional Universitaire De Tours
Department Name
Hematology
Principal Investigator Name
Emmanuel Gyan
Principal Investigator Email
E.GYAN@chu-tours.fr
Contact Person Name
Emmanuel Gyan
Contact Person Email
E.GYAN@chu-tours.fr
Site Name
Centre Hospitalier Universitaire De Rennes
Department Name
Hopital Pontchaillou - Hématologie
Principal Investigator Name
Tony MARCHAND
Principal Investigator Email
tony.marchand@chu-rennes.fr
Contact Person Name
Tony MARCHAND
Contact Person Email
tony.marchand@chu-rennes.fr
Site Name
Institut Paoli-Calmettes
Department Name
Hématologie
Principal Investigator Name
Sylvain Garciaz
Principal Investigator Email
garciazs@ipc.unicancer.fr
Contact Person Name
Sylvain Garciaz
Contact Person Email
garciazs@ipc.unicancer.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Pediatric hematology and oncology
Principal Investigator Name
PETIT Arnaud
Principal Investigator Email
arnaud.petit@aphp.fr
Contact Person Name
PETIT Arnaud
Contact Person Email
arnaud.petit@aphp.fr

Germany

Earliest CTIS Part Ii Submission Date
04-01-2024
Latest Decision Or Authorization Date
30-09-2025
Processing Time Days
636
Number Of Sites
7
Number Of Participants
25

Sites

Site Name
Universitaet Leipzig
Department Name
Med. Klinik-Poliklinik I-Hamatologie-Zelltherapie, Hamostaseologie Early Clinical Trials Unit
Principal Investigator Name
Madlen Jentzsch
Principal Investigator Email
madlen.jentzsch@medizin.uni-leipzig.de
Contact Person Name
Madlen Jentzsch
Site Name
Universitaetsklinikum Heidelberg AöR
Department Name
Medizinischen Klinik V Haematologie, Onkologie und Rheumatologie
Principal Investigator Name
Tim Sauer
Principal Investigator Email
tim.sauer@med.uni-heidelberg.de
Contact Person Name
Tim Sauer
Site Name
Technische Universitat Dresden
Department Name
Medizinische Klinik und Poliklinik I. Haematologie, Zelltherapie und medizinische Onkologie
Principal Investigator Name
Christoph Roellig
Principal Investigator Email
christoph.roellig@uniklinikum-dresden.de
Contact Person Name
Christoph Roellig
Site Name
Universitaetsklinikum Ulm AöR
Department Name
Klinik für Kinder- und Jugendmedizin; Paediatrische Onkologie
Principal Investigator Name
Miriam Erlacher
Principal Investigator Email
Miriam.Erlacher@uniklinik-ulm.de
Contact Person Name
Miriam Erlacher
Site Name
Universitaet Leipzig
Department Name
Zentrum fuer Frauen- u. Kindermedizin, Abt. fuer paediatrische Onkologie, Haematol. u. Haemostaseol
Principal Investigator Name
Lars Fischer
Principal Investigator Email
lars.fischer@medizin.uni-leipzig.de
Contact Person Name
Lars Fischer
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Medizinische Klinik m.S. Haematologie, Onkologie und Tumorimmunologie
Principal Investigator Name
Marie Luise Huetter-Kroenke
Principal Investigator Email
luise.huetter-kroenke@charite.de
Contact Person Name
Marie Luise Huetter-Kroenke
Site Name
Universitaetsklinikum Ulm AöR
Department Name
Klinik für Innere Medizin III Zentrum für Innere Medizin
Principal Investigator Name
Hartmut Doehner
Principal Investigator Email
hartmut.doehner@uniklinik-ulm.de
Contact Person Name
Hartmut Doehner

Italy

Earliest CTIS Part Ii Submission Date
04-01-2024
Latest Decision Or Authorization Date
01-10-2025
Processing Time Days
637
Number Of Sites
4
Number Of Participants
13

Sites

Site Name
Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
Department Name
Unità Operativa di Ematologia – Pad. 8
Principal Investigator Name
Cristina Papayannidis
Principal Investigator Email
cristina.papayannidis@gmail.com
Contact Person Name
Cristina Papayannidis
Site Name
Azienda Ospedaliera Ospedale Niguarda Ca Granda
Department Name
Struttura Complessa di Ematologia
Principal Investigator Name
Valeria Mancini
Principal Investigator Email
valentina.mancini@ospedaleniguarda.it
Contact Person Name
Valeria Mancini
Site Name
Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori IRST S.r.l.
Department Name
Struttura Semplice Dipartimentale (SSD) Ematologia
Principal Investigator Name
Marianna Norata
Principal Investigator Email
marianna.norata@irst.emr.it
Contact Person Name
Marianna Norata
Contact Person Email
marianna.norata@irst.emr.it
Site Name
Humanitas Research Hospital
Department Name
U.O. di Oncologia Medica ed Ematologia
Principal Investigator Name
Matteo Giovanni Della Porta
Principal Investigator Email
matteo.della_porta@hunimed.eu
Contact Person Name
Matteo Giovanni Della Porta
Contact Person Email
matteo.della_porta@hunimed.eu

Spain

Earliest CTIS Part Ii Submission Date
04-01-2024
Latest Decision Or Authorization Date
03-10-2025
Processing Time Days
639
Number Of Sites
6
Number Of Participants
30

Sites

Site Name
Clinica Universidad De Navarra
Department Name
Hematology
Principal Investigator Name
Ana Alfonso
Principal Investigator Email
aalfonso@unav.es
Contact Person Name
Ana Alfonso
Contact Person Email
aalfonso@unav.es
Site Name
Hospital Universitario Fundacion Jimenez Diaz
Department Name
Hematology
Principal Investigator Name
Gala Vega
Principal Investigator Email
gala.vega@startmadrid.com
Contact Person Name
Gala Vega
Contact Person Email
gala.vega@startmadrid.com
Site Name
Hospital Universitari Vall D Hebron
Department Name
Hematology
Principal Investigator Name
Olga Salamero
Principal Investigator Email
osalamero@vhio.net
Contact Person Name
Olga Salamero
Contact Person Email
osalamero@vhio.net
Site Name
Hospital Clinic De Barcelona
Department Name
Hematology
Principal Investigator Name
Jordi Esteve
Principal Investigator Email
jesteve@clinic.cat
Contact Person Name
Jordi Esteve
Contact Person Email
jesteve@clinic.cat
Site Name
Hospital De La Santa Creu I Sant Pau
Department Name
Hematology
Principal Investigator Name
Ana Garrido
Principal Investigator Email
AGarridoD@santpau.cat
Contact Person Name
Ana Garrido
Contact Person Email
AGarridoD@santpau.cat
Site Name
Hospital Infantil Universitario Nino Jesus
Department Name
Hematology
Principal Investigator Name
Alba Rubio
Principal Investigator Email
alba.rubio@salud.madrid.org
Contact Person Name
Alba Rubio
Contact Person Email
alba.rubio@salud.madrid.org

Sponsor

Primary sponsor

Full Name
Janssen - Cilag International
Organisation Type
Pharmaceutical company
Country Of Registered Address
Belgium

Contract research organisations

Name
IQVIA Limited
Responsibilities
code:6
Name
Parexel International (IRL) Limited
Responsibilities
code:6
Name
Ppd Inc.
Responsibilities
code:4
Name
Pharmaceutical Research Associates Group B.V.
Responsibilities
code:4
Name
4g Clinical LLC
Responsibilities
code:3

Third parties

  • {"country":"United States","full_name":"4g Clinical LLC","duties_or_roles":"code:3","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"code:6","organisation_type":"Pharmaceutical company"}
  • {"country":"Netherlands","full_name":"Pharmaceutical Research Associates Group B.V.","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Ancillare LP","duties_or_roles":"code:15; Provision of supplies for refrigerated IP transportation from site to subjects houses","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Ppd Inc.","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"code:15; Lab kits and collection services for PK samples","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Hematogenix Laboratory Services Limited","duties_or_roles":"code:15; Lab kits and collection services for PD samples","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"code:13; code:15; Central ECG analysis","organisation_type":"Pharmaceutical company"}
  • {"country":"Malaysia","full_name":"Hematogenix","duties_or_roles":"code:15; Lab kits and collection services for PD samples","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"code:6","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"eResearchTechnology GmbH","duties_or_roles":"code:13; code:15; Central ECG analysis","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
JNJ-75276617
Active Substance
BLEXIMENIB
Modality
Small molecule
Routes Of Administration
ORAL USE
Route
ORAL
Authorisation Status
1
Investigational Product Name
Cytarabine 100 mg/ml Solution for Injection or Infusion
Active Substance
CYTARABINE
Modality
Small molecule
Routes Of Administration
INTRAVENOUS USE
Route
INTRAVENOUS
Authorisation Status
2
Investigational Product Name
Idarubicin
Active Substance
IDARUBICIN
Modality
Small molecule
Routes Of Administration
INTRAVENOUS USE
Route
INTRAVENOUS
Authorisation Status
1
Investigational Product Name
Daunoblastin® (Daunorubicin)
Active Substance
DAUNORUBICIN HYDROCHLORIDE
Modality
Small molecule
Routes Of Administration
INTRAVENOUS USE
Route
INTRAVENOUS
Authorisation Status
2
Investigational Product Name
Azacitidine
Active Substance
AZACITIDINE
Modality
Small molecule
Routes Of Administration
INJECTION
Route
INJECTION
Authorisation Status
1
Investigational Product Name
Venclyxto (Venetoclax)
Active Substance
VENETOCLAX
Modality
Small molecule
Routes Of Administration
ORAL USE
Route
ORAL
Authorisation Status
2
Combination Treatment
Yes

Related trials

Other published trials that may interest you.