Clinical trial • Phase III • Neurology|Rare Disease
ONASEMNOGENE ABEPARVOVEC for Spinal muscular atrophy
Phase III trial of ONASEMNOGENE ABEPARVOVEC for Spinal muscular atrophy. open-label, none/not specified-controlled. 147 participants.
Overview
- Trial Therapeutic Area
- Neurology|Rare Disease
- Trial Disease
- Spinal muscular atrophy
- Trial Stage
- Phase III
- Drug Modality
- Gene therapy
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 10-07-2024
- First CTIS Authorization Date
- 08-08-2024
Trial design
open-label, none/not specified-controlled Phase III trial in Belgium, Denmark, France and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 147
- Trial Duration For Participant
- 1825
Eligibility
Recruits 147 paediatric patients.
- Vulnerable Population
- Vulnerable population: minors/children. The trial selects vulnerable populations (isVulnerablePopulationSelected = true). Written informed consent is required prior to any assessments and must be provided by the patient or the patient’s parent/legal guardian. Assent forms and caregiver/parent information sheets are provided for minors (country-specific assent/I CF documents listed for ages including 7-11 and 12-17; Denmark has assent documents for 12-14 and 15-17). Country-specific caregiver/parent ICFs and child/adolescent assent forms are included in the documentation.
Inclusion criteria
- {"criterion_text":"- Participated in an OAV101 clinical trial.\n- Written informed consent must be obtained before any assessment is performed.\n- Patient/Parent/legal guardian willing and able to comply with study procedures."}
Exclusion criteria
- {"criterion_text":"- There are no specific exclusion criteria for this study."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Number and proportion of patients reporting treatment-emergent serious adverse events (SAEs) by Medical Dictionary for Regulatory Activities (MedDRA) System Organ Class (SOC) and Preferred Term (PT) for the entire duration of the study (i.e., up to 5 years)","definition_or_measurement_approach":"SAEs will be summarised by MedDRA System Organ Class (SOC) and Preferred Term (PT) for the entire study duration (up to 5 years)."}
- {"endpoint_text":"- Number and proportion of patients reporting treatment-emergent adverse events of special interest (AESIs) by AESI category and MedDRA SOC and PT within each AESI category for the entire duration of the study (i.e., up to 5 years)","definition_or_measurement_approach":"AESIs will be categorised by AESI category and summarised by MedDRA SOC and PT within each AESI category for the entire study duration (up to 5 years)."}
Secondary endpoints
- {"endpoint_text":"- The number and proportion of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist","definition_or_measurement_approach":"Count and proportion reaching each milestone as per the Developmental Milestone Checklist."}
- {"endpoint_text":"- The number and proportion of participants demonstrating maintenance of each developmental milestone","definition_or_measurement_approach":"Count and proportion demonstrating maintenance of each developmental milestone (method: checklist comparison to prior status)."}
- {"endpoint_text":"- Change from Baseline in the Hammersmith Functional Motor Scale – Expanded (HFMSE) total score","definition_or_measurement_approach":"Change from baseline in total HFMSE score."}
- {"endpoint_text":"- Change from Baseline in the Revised Upper Limb Module (RULM) total score","definition_or_measurement_approach":"Change from baseline in total RULM score."}
- {"endpoint_text":"- Number and proportion of patients with potentially clinically significant vital sign findings, summarized individually for each vital sign parameter","definition_or_measurement_approach":"Number and proportion with clinically significant abnormal vital sign findings, summarized by each vital sign parameter."}
- {"endpoint_text":"- Number and proportion of patients with potentially clinically significant laboratory values, summarized individually for each laboratory parameter","definition_or_measurement_approach":"Number and proportion with clinically significant laboratory abnormalities, summarized by each laboratory parameter."}
Recruitment
- Planned Sample Size
- 147
- Recruitment Window Months
- 92
- Consent Approach
- Written informed consent must be obtained before any assessment. Consent may be provided by the patient or by the parent/legal guardian where applicable. Assent is used for minors: country-specific assent documents are provided (examples in the documentation include assent forms for ages 7-11 and 12-17 in Belgium; assent forms for 12-14 and 15-17 in Denmark; child and adolescent assent in Italy; assent documents in the Netherlands). Caregiver/parent information sheets and caregiver consent forms are provided. Country-specific ICF and assent documents are available in the listed country languages.
Geography
- Total Number Of Sites
- 9
- Total Number Of Participants
- 26
Belgium
- Earliest CTIS Part Ii Submission Date
- 27-06-2024
- Latest Decision Or Authorization Date
- 08-08-2024
- Processing Time Days
- 42
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- UZ Leuven
- Department Name
- 1201: Pediatric neurology
- Principal Investigator Name
- Liesbeth De Waele
- Principal Investigator Email
- liesbeth.dewaele@uzleuven.be
- Contact Person Name
- Liesbeth De Waele
- Contact Person Email
- liesbeth.dewaele@uzleuven.be
- Number Of Participants
- 4
Denmark
- Earliest CTIS Part Ii Submission Date
- 27-06-2024
- Latest Decision Or Authorization Date
- 13-08-2024
- Processing Time Days
- 47
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Rigshospitalet
- Department Name
- 1701: Hematology
- Principal Investigator Name
- Alfred Peter Born
- Principal Investigator Email
- alfred.peter.born@regionh.dk
- Contact Person Name
- Alfred Peter Born
- Contact Person Email
- alfred.peter.born@regionh.dk
- Number Of Participants
- 3
France
- Earliest CTIS Part Ii Submission Date
- 27-06-2024
- Latest Decision Or Authorization Date
- 20-08-2024
- Processing Time Days
- 54
- Number Of Sites
- 4
- Number Of Participants
- 7
Sites
- Site Name
- Les Hopitaux Universitaires De Strasbourg
- Department Name
- 1902: Département de Pédiatrie
- Principal Investigator Name
- Vincent Laugel
- Principal Investigator Email
- vincent.laugel@chru-strasbourg.fr
- Contact Person Name
- Vincent Laugel
- Contact Person Email
- vincent.laugel@chru-strasbourg.fr
- Number Of Participants
- 7
- Site Name
- Raymond-Poincare Hospital
- Department Name
- 1901: Unité Neuromusculaire, Département de Neurologie pédiatrique et Unité de Soins Intensifs
- Principal Investigator Name
- Susana Quijano-Roy
- Principal Investigator Email
- susana.quijano-roy@aphp.fr
- Contact Person Name
- Susana Quijano-Roy
- Contact Person Email
- susana.quijano-roy@aphp.fr
- Site Name
- Hospital Femme Mere Enfant
- Department Name
- 1903: Service MPR pédiatrique L'Escale
- Principal Investigator Name
- Laure Le Goff
- Principal Investigator Email
- laure.le-goff@chu-lyon.fr
- Contact Person Name
- Laure Le Goff
- Contact Person Email
- laure.le-goff@chu-lyon.fr
- Site Name
- Hopital Des Enfants
- Department Name
- 1904: Neurologie pédiatrique
- Principal Investigator Name
- Claude Cancès
- Principal Investigator Email
- cances.c@chu-toulouse.fr
- Contact Person Name
- Claude Cancès
- Contact Person Email
- cances.c@chu-toulouse.fr
Italy
- Earliest CTIS Part Ii Submission Date
- 27-06-2024
- Latest Decision Or Authorization Date
- 17-09-2024
- Processing Time Days
- 82
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Universita Cattolica Del Sacro Cuore
- Department Name
- 2201: Pediatric neurology
- Principal Investigator Name
- Eugenio Mercuri
- Principal Investigator Email
- eugeniomaria.mercuri@policlinicogemelli.it
- Contact Person Name
- Eugenio Mercuri
- Contact Person Email
- eugeniomaria.mercuri@policlinicogemelli.it
- Number Of Participants
- 3
Spain
- Earliest CTIS Part Ii Submission Date
- 27-06-2024
- Latest Decision Or Authorization Date
- 26-08-2024
- Processing Time Days
- 60
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- 3201: Pediatric Neurology
- Principal Investigator Name
- Francina Munell Casadesus
- Principal Investigator Email
- francina.munell@vhir.org
- Contact Person Name
- Francina Munell Casadesus
- Contact Person Email
- francina.munell@vhir.org
- Number Of Participants
- 3
Netherlands
- Earliest CTIS Part Ii Submission Date
- 27-06-2024
- Latest Decision Or Authorization Date
- 12-08-2024
- Processing Time Days
- 46
- Number Of Sites
- 1
- Number Of Participants
- 6
Sites
- Site Name
- Universitair Medisch Centrum Utrecht
- Department Name
- 2601: Sylvia Toth Centrum
- Principal Investigator Name
- W.L. (Ludo) van der Pol
- Principal Investigator Email
- W.L.vanderPol@umcutrecht.nl
- Contact Person Name
- W.L. (Ludo) van der Pol
- Contact Person Email
- W.L.vanderPol@umcutrecht.nl
- Number Of Participants
- 6
Sponsor
Primary sponsor
- Full Name
- Novartis Pharma AG
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Switzerland
Contract research organisations
- Name
- Parexel International (IRL) Limited
- Responsibilities
- Study Start-Up (including site contracts and Ethics committee submissions) Site Management; duties codes:1,10,11,12,15,5,6,7,8
- Name
- Q Squared Solutions Holdings LLC
- Responsibilities
- code:4
- Name
- Q Squared Solutions Limited
- Responsibilities
- code:4
- Name
- Labcorp Early Development Laboratories Inc.
- Responsibilities
- code:4
- Name
- Bioagilytix Labs LLC
- Responsibilities
- code:4
- Name
- Eresearchtechnology Inc.
- Responsibilities
- PRO management, PRO licensing and translations, data collection via tablet, Site Training for clinical evaluators.
- Name
- SGS France
- Responsibilities
- code:4
Third parties
- {"country":"Germany","full_name":"ProtaGene CGT GmbH","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Q Squared Solutions Holdings LLC","duties_or_roles":"code:4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Transperfect Translations International Inc.","duties_or_roles":"Translation and licensing","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Labcorp Early Development Laboratories Inc.","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Transport/ Concierge services for patients","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"code:4","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Ancillare LP","duties_or_roles":"Ordering, storage and distribution of RULM kits","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"codes:1,10,11,12,15,5,6,7,8; Study Start-Up (including site contracts and Ethics committee submissions) Site Management","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"PRO management, PRO licensing and translations, data collection via tablet, Site Training for clinical evaluators.","organisation_type":"Pharmaceutical company"}
- {"country":"France","full_name":"SGS France","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- OAV101B
- Active Substance
- ONASEMNOGENE ABEPARVOVEC
- Modality
- Gene therapy
- Routes Of Administration
- Intrathecal
- Route
- Intrathecal
- Orphan Designation
- Yes
- Investigational Product Name
- Zolgensma 2 × 10^13 vector genomes/mL solution for infusion
- Active Substance
- ONASEMNOGENE ABEPARVOVEC
- Modality
- Gene therapy
- Routes Of Administration
- Intravenous
- Route
- Intravenous
- Authorisation Status
- Authorised (EU marketing authorisation EU/1/20/1443/001)
- Orphan Designation
- Yes
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