Clinical trial • Phase I/II • Oncology
ASCIMINIB HYDROCHLORIDE for Philadelphia chromosome-positive chronic myeloid leukemia (chronic phase)|Juvenile chronic myeloid leukemia
Phase I/II trial of ASCIMINIB HYDROCHLORIDE for Philadelphia chromosome-positive chronic myeloid leukemia (chronic phase)|Juvenile chronic myeloid leukemi…
Overview
- Trial Therapeutic Area
- Oncology
- Trial Disease
- Philadelphia chromosome-positive chronic myeloid leukemia (chronic phase)|Juvenile chronic myeloid leukemia
- Trial Stage
- Phase I/II
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 17-04-2024
- First CTIS Authorization Date
- 28-05-2024
Trial design
open-label, none/not specified-controlled Phase I/II trial in Netherlands, Germany, France and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Single Multiple Or Escalation Dose Combined
- Yes
- Target Sample Size
- 23
Eligibility
Recruits 23 paediatric patients.
- Vulnerable Population
- The trial enrols pediatric participants (age groups include ≥1 to <18 years; with specific adult-formulation group for 14–<18 years with ≥40 kg). Assent is obtained from children/adolescents (child assent, adolescent assent forms) and informed consent is obtained from parent/legal guardian (parent/legal guardian ICFs). Age-specific ICFs (child, pre-adolescent, adolescent, adolescent becoming adult) and separate data protection consent forms are included; country/language-specific ICFs are provided.
Inclusion criteria
- {"criterion_text":"- Male or female participants: a. Pediatric formulation group: ≥1 and less than 18 years of age at study entry. b. Adult formulation group: ≥14 and less than 18 years of age and body weight of ≥ 40 kg at study entry.\n- Participants with Ph+ CML-CP must meet all of the following laboratory values at the screening visit. In the case where bone marrow blast and promyelocyte counts are available, these will be accepted if done locally within 56 days prior to the screening visit, to avoid unnecessary repetition of this test. a. <15% blasts in peripheral blood and bone marrow b. < 30% combined blasts plus promyelocytes in peripheral blood and bone marrow c. < 20% basophils in the peripheral blood d. Neutrophils ≥ 1.5 x 10^9/L (or white blood cell (WBC) ≥ 3 x 10^9/L if neutrophils are not available) and platelet count ≥ 100 x 10^9/L e. No evidence of extramedullary leukemic involvement, with the exception of hepatosplenomegaly\n- Prior treatment with a minimum of one TKI.\n- Failure or intolerance to the most recent TKI therapy at the time of screening.\n- Evidence of typical BCR-ABL fusion gene (BCR-ABL1) transcript [e14a2 and/or e13a2] at the time of screening which are amenable to standardized real time quantitative polymerase chain reaction (RQ-PCR) quantification."}
Exclusion criteria
- {"criterion_text":"- Known presence of the T315I mutation prior to study entry or of a BCR: ABL mutation with known resistance to study treatment any time prior to study entry\n- Known second chronic phase of CML after previous progression to AP/BC.\n- Previous treatment with a hematopoietic stem-cell transplantation.\n- Patient planning to undergo allogeneic hematopoietic stem cell transplantation.\n- Cardiac or cardiac repolarization abnormality."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Primary PK parameters of asciminib: AUClast, AUCtau","definition_or_measurement_approach":"Pharmacokinetic parameters derived from plasma concentration–time profiles (AUClast, AUCtau); PK sampling/assay details not provided in the JSON."}
- {"endpoint_text":"- Secondary PK parameters of asciminib: Cmax, Tmax, Ctrough.","definition_or_measurement_approach":"Pharmacokinetic parameters derived from plasma concentration–time profiles (Cmax, Tmax, Ctrough); specific assay/methodology not detailed in the JSON."}
Secondary endpoints
- {"endpoint_text":"- Number, seriousness, severity, and causality assessments of treatment-emergent adverse events and other safety data as considered appropriate.","definition_or_measurement_approach":"Safety assessed by recording treatment-emergent adverse events, with standard assessments of seriousness, severity and causality; further safety assessment details not provided in the JSON."}
- {"endpoint_text":"- Activity: Hematologic and molecular responses.","definition_or_measurement_approach":"Hematologic and molecular responses assessed (molecular response likely via standardized RQ-PCR of BCR-ABL transcripts as referenced elsewhere), specific response criteria not detailed in the JSON."}
- {"endpoint_text":"- Questionnaire on acceptability and palatability after first dose, 4 and 52 weeks.","definition_or_measurement_approach":"Patient/caregiver-reported questionnaire administered after first dose, at 4 weeks and at 52 weeks to assess acceptability and palatability."}
- {"endpoint_text":"- Number, seriousness, severity, and causality assessments of treatment-emergent adverse events and other safety data as considered appropriate including growth and sexual maturation assessments.","definition_or_measurement_approach":"Safety assessments as above with additional specific assessments of growth and sexual maturation; measurement details not specified in the JSON."}
Recruitment
- Planned Sample Size
- 23
- Recruitment Window Months
- 108
- Consent Approach
- Informed consent is obtained from the parent/legal guardian (Parent Legal Guardian ICFs); assent is obtained from children/adolescents (Child Assent, Adolescent Assent forms). There are age-specific ICFs including adolescent becoming adult and separate data protection consent forms. Country- and language-specific ICFs are supplied (examples in Polish, Hungarian, Dutch, Greek, Italian, German, French and English in the submitted documents).
Geography
- Total Number Of Sites
- 15
- Total Number Of Participants
- 17
Netherlands
- Earliest CTIS Part Ii Submission Date
- 28-05-2024
- Latest Decision Or Authorization Date
- 10-02-2026
- Processing Time Days
- 623
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Prinses Maxima Centrum voor Kinderoncologie B.V.
- Department Name
- #1101:Kinderoncologie
- Principal Investigator Name
- Michel Zwaan
- Principal Investigator Email
- c.m.zwaan@prinsesmaximacentrum.nl
- Contact Person Name
- Michel Zwaan
- Contact Person Email
- c.m.zwaan@prinsesmaximacentrum.nl
Germany
- Earliest CTIS Part Ii Submission Date
- 29-05-2024
- Latest Decision Or Authorization Date
- 11-03-2026
- Processing Time Days
- 651
- Number Of Sites
- 3
- Number Of Participants
- 3
Sites
- Site Name
- University Medical Center Hamburg-Eppendorf
- Department Name
- #1502: Klinik für pädiatrische Hämatologie und Onkologie
- Principal Investigator Name
- Gabriele Escherich
- Principal Investigator Email
- escherich@uke.de
- Contact Person Name
- Gabriele Escherich
- Contact Person Email
- escherich@uke.de
- Site Name
- Universitaetsklinikum Erlangen AöR
- Department Name
- #1501: Kinder und Jugendklinik
- Principal Investigator Name
- Markus Metzler
- Principal Investigator Email
- Markus.metzler@uk-erlangen.de
- Contact Person Name
- Markus Metzler
- Contact Person Email
- Markus.metzler@uk-erlangen.de
- Site Name
- Universitaetsklinikum Essen AöR
- Department Name
- #1504: Zentrum für Kinder- und Jugendmedizin
- Principal Investigator Name
- Michaela Höfs
- Principal Investigator Email
- michaela.hoefs@uk-essen.de
- Contact Person Name
- Michaela Höfs
- Contact Person Email
- michaela.hoefs@uk-essen.de
France
- Earliest CTIS Part Ii Submission Date
- 03-06-2024
- Latest Decision Or Authorization Date
- 16-03-2026
- Processing Time Days
- 651
- Number Of Sites
- 4
- Number Of Participants
- 3
Sites
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- #1202:Unité d’Hematologie Pediatrique
- Principal Investigator Name
- Brigitte NELKEN
- Principal Investigator Email
- brigitte.nelker@chru-lille.fr
- Contact Person Name
- Brigitte NELKEN
- Contact Person Email
- brigitte.nelker@chru-lille.fr
- Site Name
- Centre Hospitalier Universitaire De Poitiers
- Department Name
- #1201:Service d’Hematologie Oncologie Pediatrique
- Principal Investigator Name
- Frederic MILLOT
- Principal Investigator Email
- f.millot@chu-poitiers.fr
- Contact Person Name
- Frederic MILLOT
- Contact Person Email
- f.millot@chu-poitiers.fr
- Site Name
- Centre Hospitalier Universitaire De Bordeaux
- Department Name
- #1203:Unite d’Hematologie Oncologie Pediatrique
- Principal Investigator Name
- Charlotte JUBERT
- Principal Investigator Email
- charlotte.jubert@chu-bordeaux.fr
- Contact Person Name
- Charlotte JUBERT
- Contact Person Email
- charlotte.jubert@chu-bordeaux.fr
- Site Name
- Robert Debre University Hospital
- Department Name
- #1204:Service d’Hematologie-Immunologie Pediatrique
- Principal Investigator Name
- Benoit BRETHON
- Principal Investigator Email
- benoit.brethon@aphp.fr
- Contact Person Name
- Benoit BRETHON
- Contact Person Email
- benoit.brethon@aphp.fr
Italy
- Earliest CTIS Part Ii Submission Date
- 09-07-2024
- Latest Decision Or Authorization Date
- 10-02-2026
- Processing Time Days
- 581
- Number Of Sites
- 4
- Number Of Participants
- 5
Sites
- Site Name
- Ospedale Pediatrico Bambino Gesu
- Department Name
- #7004:Dipartimento Ematologia Oncologia e Medicina Trasfusionale
- Principal Investigator Name
- Franco LOCATELLI
- Principal Investigator Email
- franco.locatelli@opbg.net
- Contact Person Name
- Franco LOCATELLI
- Contact Person Email
- franco.locatelli@opbg.net
- Site Name
- Giannina Gaslini Institute For Scientific Hospitalization And Care
- Department Name
- #7001:U.O.C. Ematologia
- Principal Investigator Name
- Elena PALMISANI
- Principal Investigator Email
- elenapalmisani@gaslini.org
- Contact Person Name
- Elena PALMISANI
- Contact Person Email
- elenapalmisani@gaslini.org
- Site Name
- Fondazione IRCCS San Gerardo Dei Tintori
- Department Name
- #7002:Clinica Pediatrica
- Principal Investigator Name
- Adriana Cristina BALDUZZI
- Principal Investigator Email
- abalduzzi@fondazionembbm.it
- Contact Person Name
- Adriana Cristina BALDUZZI
- Contact Person Email
- abalduzzi@fondazionembbm.it
- Site Name
- Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
- Department Name
- #7003:S.C. Oncoematologia Pediatrica Presidio Ospedale Infantile Regina Margherita
- Principal Investigator Name
- Franca FAGIOLI
- Principal Investigator Email
- franca.fagioli@unito.it
- Contact Person Name
- Franca FAGIOLI
- Contact Person Email
- franca.fagioli@unito.it
Poland
- Earliest CTIS Part Ii Submission Date
- 05-07-2024
- Latest Decision Or Authorization Date
- 16-02-2026
- Processing Time Days
- 591
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
- Department Name
- #1401:Klinika Transplantologii Szpiku, Onkologii i Hematologii Dzięcięcej
- Principal Investigator Name
- Krzysztof Kałwak
- Principal Investigator Email
- Krzysztof.kalwak@gmail.com
- Contact Person Name
- Krzysztof Kałwak
- Contact Person Email
- Krzysztof.kalwak@gmail.com
Hungary
- Earliest CTIS Part Ii Submission Date
- 04-06-2024
- Latest Decision Or Authorization Date
- 13-02-2026
- Processing Time Days
- 619
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Semmelweis University
- Department Name
- 9001:Tuzolto utcai reszleg
- Principal Investigator Name
- Judit Muller
- Principal Investigator Email
- muller.judit@med.semmelweis-univ.hu
- Contact Person Name
- Judit Muller
- Contact Person Email
- muller.judit@med.semmelweis-univ.hu
Greece
- Earliest CTIS Part Ii Submission Date
- 07-08-2024
- Latest Decision Or Authorization Date
- 11-02-2026
- Processing Time Days
- 553
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Nosokomeio Paidon I Agia Sofia
- Department Name
- #1301:1st Pediatric Clinic
- Principal Investigator Name
- Antonis Kattamis
- Principal Investigator Email
- ankatt@med.uoa.gr
- Contact Person Name
- Antonis Kattamis
- Contact Person Email
- ankatt@med.uoa.gr
Sponsor
Primary sponsor
- Full Name
- Novartis Pharma AG
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Switzerland
Contract research organisations
- Name
- IQVIA Limited
- Responsibilities
- code 3; code 1 (various roles listed across entries)
- Name
- Syneos Health Inc.
- Responsibilities
- code 1
- Name
- Icon Clinical Research Limited
- Responsibilities
- code 1; code 4
- Name
- Parexel International (IRL) Limited
- Responsibilities
- code 12
- Name
- PRA Hellas CRO A.E.
- Responsibilities
- code 1
- Name
- Q Squared Solutions Limited
- Responsibilities
- code 4
- Name
- Labcorp Pharmaceutical Research And Development (Shanghai) Co. Ltd.
- Responsibilities
- code 4
- Name
- Veeda Clinical Research Limited
- Responsibilities
- code 4
Third parties
- {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"code 4","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"code 3","organisation_type":"Pharmaceutical company"}
- {"country":"France","full_name":"Creapharm Clinical Supplies","duties_or_roles":"code 14; code 15 (Drug return and destruction)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"code 1","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"code 1","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"code 1","organisation_type":"Pharmaceutical company"}
- {"country":"Greece","full_name":"PRA Hellas CRO A.E.","duties_or_roles":"code 1","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"code 12","organisation_type":"Pharmaceutical company"}
- {"country":"Poland","full_name":"Eco-Abc Sp. z o. o.","duties_or_roles":"code 15 (Destruction of the investigational medicinal products)","organisation_type":"Pharmaceutical company"}
- {"country":"Poland","full_name":"Statmed Sp. z o.o.","duties_or_roles":"code 15 (Compensation for patients travel to the clinical site)","organisation_type":"Pharmaceutical company"}
- {"country":"Italy","full_name":"Opis S.r.l.","duties_or_roles":"code 15 (TMF archive)","organisation_type":"Pharmaceutical company"}
- {"country":"Hungary","full_name":"ADR Logistics Kft.","duties_or_roles":"code 15 (Drug storage, distribution, and destruction)","organisation_type":"Pharmaceutical company"}
- {"country":"Italy","full_name":"Mipharm S.p.A.","duties_or_roles":"code 15 (Local equipment storage)","organisation_type":"Pharmaceutical company"}
- {"country":"China","full_name":"Labcorp Pharmaceutical Research And Development (Shanghai) Co. Ltd.","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
- {"country":"India","full_name":"Veeda Clinical Research Limited","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Asciminib
- Active Substance
- ASCIMINIB HYDROCHLORIDE
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL
- Authorisation Status
- prodAuthStatus 1
- Orphan Designation
- Yes
- Investigational Product Name
- ASCIMINIB
- Active Substance
- ASCIMINIB
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL
- Authorisation Status
- prodAuthStatus 2
- Orphan Designation
- Yes
- Investigational Product Name
- ASCIMINIB HYDROCHLORIDE
- Active Substance
- ASCIMINIB HYDROCHLORIDE
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL
- Authorisation Status
- prodAuthStatus 2
- Orphan Designation
- Yes
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