Clinical trial • Phase III • Musculoskeletal
Delandistrogene moxeparvovec for Duchenne muscular dystrophy
Phase III trial of Delandistrogene moxeparvovec for Duchenne muscular dystrophy. open-label, none/not specified-controlled. 319 participants.
Overview
- Trial Therapeutic Area
- Musculoskeletal
- Trial Disease
- Duchenne muscular dystrophy
- Trial Stage
- Phase III
- Drug Modality
- Gene therapy
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 17-11-2023
- First CTIS Authorization Date
- 14-03-2024
Trial design
open-label, none/not specified-controlled Phase III trial across 7 sites in Belgium, Germany, Italy and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 319
- Trial Duration For Participant
- 3435
Eligibility
Recruits 319 paediatric patients.
- Vulnerable Population
- The trial includes vulnerable populations (minors). Consent/assent handling: consent is provided by a parent(s) or legal caregiver(s) for participants who are minors; participants ≥18 provide their own consent. Country-specific assent and consent documents are available (multiple country ICF and assent forms listed for ages/strata such as assent for ages 6-11, 7-12, 13-17). Language-specific ICF/assent documents are provided (English, Italian, German, Spanish, French, Dutch as per country documents).
Inclusion criteria
- {"criterion_text":"- Received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a previous clinical study"}
- {"criterion_text":"- Has (a) parent(s) or legal caregiver(s) or is ≥18 years of age and able to understand and comply with the study visit schedule and all other protocol requirements"}
Exclusion criteria
- {"criterion_text":"-"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)","definition_or_measurement_approach":""}
Secondary endpoints
- {"endpoint_text":"- Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change in Time to Rise From Floor From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change in Time of 10-meter Walk/Run (10MWR) from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation.","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change in PUL (Version 2.0) Domain Specific Scores from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change in Forced Vital Capacity Percent (FVC%) Predicted from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change in Peak Expiratory Flow Percent (PEF%) Predicted from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change in Cardiac Magnetic Resonance Imaging (MRI) findings from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change in Musculoskeletal MRI Findings from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation","definition_or_measurement_approach":""}
Recruitment
- Planned Sample Size
- 319
- Recruitment Window Months
- 112
- Consent Approach
- Informed consent is obtained from participants ≥18 years. For minors, consent is obtained from a parent(s) or legal caregiver(s) and assent is obtained from the child as appropriate. Age-specific assent/ICF documents are provided (examples: Italy assent 6-11y and 12-17y; Germany assent 7-12y and 13-17y; Belgium assent 7-12y and 13-maturity; Spain and other countries have country-specific ICF and assent forms). Documents are available in multiple languages (English, Italian, German, Spanish, French, Dutch) as per country.
Methods
- Patient recruitment via SareptAlly contact (email: SareptAlly@sarepta.com; telephone: +18887273782)
- Site-based recruitment at participating hospital/clinic sites in Belgium, Germany, Italy, and Spain (local site investigators and clinics listed in country Part II entries)
- Country-specific recruitment procedures documented in K1 recruitment arrangements (country K1 recruitment procedure description documents available for Belgium, Germany, Italy, Spain)
Geography
- Total Number Of Sites
- 7
- Total Number Of Participants
- 74
Belgium
- Earliest CTIS Part Ii Submission Date
- 22-02-2024
- Latest Decision Or Authorization Date
- 14-04-2026
- Processing Time Days
- 782
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Universitair Ziekenhuis Gent
- Department Name
- 406: Neuromuscular Reference Centre (NMRC)
- Principal Investigator Name
- Nicolas Deconinck
- Principal Investigator Email
- nicolas.deconinck@huderf.be
- Contact Person Name
- Nicolas Deconinck
- Contact Person Email
- nicolas.deconinck@huderf.be
Germany
- Earliest CTIS Part Ii Submission Date
- 29-01-2024
- Latest Decision Or Authorization Date
- 14-04-2026
- Processing Time Days
- 806
- Number Of Sites
- 1
- Number Of Participants
- 9
Sites
- Site Name
- Universitaetsklinikum Essen AöR
- Department Name
- 409: Klinik für Kinderheilkunde I
- Principal Investigator Name
- Andrea Gangfuss
- Principal Investigator Email
- andrea.gangfuss@uk-essen.de
- Contact Person Name
- Andrea Gangfuss
- Contact Person Email
- andrea.gangfuss@uk-essen.de
Italy
- Earliest CTIS Part Ii Submission Date
- 20-02-2024
- Latest Decision Or Authorization Date
- 20-04-2026
- Processing Time Days
- 790
- Number Of Sites
- 3
- Number Of Participants
- 24
Sites
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- 404: U.O.C. Neuropsichiatria Infantile
- Principal Investigator Name
- Eugenio Mercuri
- Principal Investigator Email
- eugeniomaria.mercuri@policlinicogemelli.it
- Contact Person Name
- Eugenio Mercuri
- Contact Person Email
- eugeniomaria.mercuri@policlinicogemelli.it
- Site Name
- Giannina Gaslini Institute For Scientific Hospitalization And Care
- Department Name
- 416: Centro di Miologia Traslazionale e Sperimentale
- Principal Investigator Name
- Claudio Bruno
- Principal Investigator Email
- claudiobruno@gaslini.org
- Contact Person Name
- Claudio Bruno
- Contact Person Email
- claudiobruno@gaslini.org
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- 444: UOC Neurologia Pad. Monteggia
- Principal Investigator Name
- Giacomo Pietro Comi
- Principal Investigator Email
- giacomo.comi@unimi.it
- Contact Person Name
- Giacomo Pietro Comi
- Contact Person Email
- giacomo.comi@unimi.it
Spain
- Earliest CTIS Part Ii Submission Date
- 22-02-2024
- Latest Decision Or Authorization Date
- 20-04-2026
- Processing Time Days
- 788
- Number Of Sites
- 2
- Number Of Participants
- 36
Sites
- Site Name
- Sant Joan De Deu Barcelona Hospital
- Department Name
- 425: Neuropediatría
- Principal Investigator Name
- Andres Nascimento Osorio
- Principal Investigator Email
- andres.nascimento@sjd.es
- Contact Person Name
- Andres Nascimento Osorio
- Contact Person Email
- andres.nascimento@sjd.es
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- 424: Neurología
- Principal Investigator Name
- Nuria Muelas Gómez
- Principal Investigator Email
- nuriamugo@gmail.com
- Contact Person Name
- Nuria Muelas Gómez
- Contact Person Email
- nuriamugo@gmail.com
Sponsor
Primary sponsor
- Full Name
- Sarepta Therapeutics Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Parexel International (IRL) Limited
- Responsibilities
- Site Management, Site Contract/Budget Negotiation, Import/Export, Equipment Provisioning
Third parties
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"Site Management, Site Contract/Budget Negotiation, Import/Export, Equipment Provisioning","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Delandistrogene moxeparvovec-rokl
- Active Substance
- Delandistrogene moxeparvovec
- Modality
- Gene therapy
- Routes Of Administration
- Intravenous
- Route
- Intravenous
- Authorisation Status
- prodAuthStatus:1
- Orphan Designation
- Yes
- Maximum Dose
- 13300000000000 vector genomes (vg)/mL
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