Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ongoing, recruiting
Participants planned
393
Countries
4
Sites
7
Duchenne Muscular Dystrophy
To evaluate the long-term safety of delandistrogene moxeparvovec
Key facts
- Sponsor
- Sarepta Therapeutics Inc.
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years
- Gender
- Male
- Therapeutic area
- Diseases [C] - Musculoskeletal Diseases [C05]
- Trial duration
- 24 May 2024 → ongoing
- Decision date (initial)
- 2024-03-19
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Sarepta Therapeutics Inc.
External identifiers
- EU CT number
- 2023-505043-39-00
- WHO UTN
- U1111-1296-0489
- ClinicalTrials.gov
- NCT05967351
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Safety
To evaluate the long-term safety of delandistrogene moxeparvovec
Secondary objectives 1
- To evaluate the long-term efficacy of delandistrogene moxeparvovec
Conditions and MedDRA coding
Duchenne Muscular Dystrophy
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10013801 | Duchenne muscular dystrophy | 100000004850 |
| 20.1 | PT | 10052655 | Duchenne muscular dystrophy gene carrier | 100000004850 |
Regulatory references
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2020-002372-13 | A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION) , Étude de phase 3, internationale, randomisée, en double aveugle, contrôlée contre placebo, de thérapie génique systémique, destinée à évaluer la sécurité d’emploi et l’efficacité du SRP9001 chez des patients non ambulatoires et ambulatoires atteints de dystrophie musculaire de Duchenne (ENVISION), Studio di fase 3, internazionale, randomizzato, in doppio cieco, controllato verso placebo, sulla terapia di trasferimento sistemico di geni, per valutare la sicurezza e l’efficacia di SRP-9001 in soggetti deambulanti e non deambulanti con distrofia muscolare di Duchenne (ENVISION), Estudio de fase 3, multinacional, aleatorizado, en doble ciego y controlado con placebo, del tratamiento mediante transferencia génica para evaluar la seguridad y la eficacia de SRP-9001 en sujetos, no ambulatorios y ambulatorios, con distrofia muscular de Duchenne (ENVISION) | |
| 2022-000691-19 | A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchenne muscular dystrophy, Estudio abierto, de dos partes, de administración génica sistémica para evaluar la seguridad y la expresión de RO7494222 (SRP-9001) en sujetos menores de cuatro años con Distrofia Muscular de Duchenne, Uno studio in due parti, in aperto, sulla terapia genica sistemica per valutare la sicurezza e l’espressione di RO7494222 (SRP-9001) in soggetti di età inferiore a quattro anni affetti da distrofia muscolare di Duchenne, Etude en 2 parties, en ouvert, de transfert de gène par voir systémique, évaluant la sécurité et l'expression de RO7494222 (SRP-9001) chez l'enfant âgé de moins de quatre ans présentant une dystrophie musculaire de Duchenne | |
| 2019-003374-91 | A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK) , Étude de phase 3, multinationale, randomisée, en double aveugle, contrôlée par placebo, sur le transfert systémique de gènes, visant à évaluer la sécurité d’emploi et l’efficacité du SRP-9001 chez des sujets atteints de dystrophie musculaire de Duchenne (EMBARK), Estudio de fase III multinacional, aleatorizado, doble ciego, controlado con placebo, de liberación génica sistémica para evaluar la seguridad y la eficacia de SRP-9001 en sujetos con distrofia muscular de Duchenne (EMBARK), Studio di fase 3, internazionale, randomizzato, in doppio cieco, controllato verso placebo, sul rilascio sistemico di geni, per valutare la sicurezza e l’efficacia di SRP-9001 in soggetti con distrofia muscolare di Duchenne (EMBARK) |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- Received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a previous clinical study
- 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 1
- -
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
Secondary endpoints 9
- Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation
- Change in Time to Rise From Floor From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation
- Change in Time of 10-meter Walk/Run (10MWR) from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation.
- 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
- Change in PUL (Version 2.0) Domain Specific Scores from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation
- Change in Forced Vital Capacity Percent (FVC%) Predicted from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation
- Change in Peak Expiratory Flow Percent (PEF%) Predicted from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation
- Change in Cardiac Magnetic Resonance Imaging (MRI) findings from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation
- Change in Musculoskeletal MRI Findings from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Delandistrogene moxeparvovec-rokl
PRD8656851 · Product
- Active substance
- Delandistrogene Moxeparvovec
- Pharmaceutical form
- SOLUTION FOR INJECTION/INFUSION
- Route of administration
- INTRAVENOUS USE
- Max daily dose
- 13300000000000 vector genomes (vg)/mL
- Max total dose
- 13300000000000 vector genomes (vg)/mL
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Not Authorised
- MA holder
- SAREPTA THERAPEUTICS INC
- Paediatric formulation
- Yes
- Orphan designation
- Yes
- Orphan designation number
- EU/3/20/2250
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Sarepta Therapeutics Inc.
- Sponsor organisation
- Sarepta Therapeutics Inc.
- Address
- 215 1st Street
- City
- Cambridge
- Postcode
- 02142-1213
- Country
- United States
Scientific contact point
- Organisation
- Sarepta Therapeutics Inc.
- Contact name
- Sarepta Therapeutics Inc.
Public contact point
- Organisation
- Sarepta Therapeutics Inc.
- Contact name
- Patient Recruitment
Third parties 1
| Organisation | City, country | Duties |
|---|---|---|
| Parexel International (IRL) Limited ORG-100022780
|
Dublin 2, Ireland | On site monitoring, Code 12, Code 13, Other, Code 5, Data management, E-data capture, Code 8 |
Locations
4 EU/EEA countries · 7 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Belgium | Ongoing, recruiting | 5 | 1 |
| Germany | Ongoing, recruiting | 9 | 1 |
| Italy | Ongoing, recruiting | 24 | 3 |
| Spain | Ongoing, recruiting | 36 | 2 |
| Rest of world
Australia, United States, United Kingdom, Israel, Hong Kong, Taiwan, Japan
|
— | 319 | — |
Investigational sites
Universitair Ziekenhuis Gent
406: Neuromuscular Reference Centre (NMRC), Corneel Heymanslaan 10, 9000, Gent
Universitaetsklinikum Essen AöR
409: Klinik für Kinderheilkunde I, Hufelandstrasse 55, Holsterhausen, Essen
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
404: U.O.C. Neuropsichiatria Infantile, Largo Francesco Vito 1, 00168, Rome
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
444: UOC Neurologia Pad. Monteggia, Via Francesco Sforza 28, 20122, Milan
Giannina Gaslini Institute For Scientific Hospitalization And Care
416: Centro di Miologia Traslazionale e Sperimentale, Via Gerolamo Gaslini 5, 16147, Genoa
Hospital Universitario Y Politecnico La Fe
424: Neurología, Avenida De Fernando Abril Martorell 106, 46026, Valencia
Sant Joan De Deu Barcelona Hospital
425: Neuropediatría, Passeig De Sant Joan De Deu 2, 08950, Esplugues De Llobregat
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Belgium | 2024-09-26 | 2024-10-16 | |||
| Germany | 2024-08-26 | 2024-09-23 | |||
| Italy | 2024-09-02 | 2024-09-04 | |||
| Spain | 2024-05-24 | 2024-05-28 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 57 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol Main English SRP-9001-305 Public | 2.0 |
| Protocol (for publication) | D4_Subject Questionnaire 1 Dutch SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 1 French SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 1 German_BEL SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 1 German_DEU SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 1 Italian SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 1 Spanish SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 2 Dutch SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 2 French SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 2 German_BEL SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 2 German_DEU SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 2 Italian SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 2 Spanish SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 3 Dutch SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 3 French SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 3 German_BEL SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 3 German_DEU SRP-9001-305 Public | 1.0 |
| Protocol (for publication) | D4_Subject Questionnaire 3 Italian SRP-9001-305 Public | 1.1 |
| Protocol (for publication) | D4_Subject Questionnaire 3 Spanish SRP-9001-305 Public | 1.0 |
| Recruitment arrangements (for publication) | K1_BEL Recruitment Procedure Description English SRP-9001-305 Public | 1.0 |
| Recruitment arrangements (for publication) | K1_DEU Recruitment Procedure Description English SRP-9001-305 Public | 1.0 |
| Recruitment arrangements (for publication) | K1_ESP Recruitment Procedure Description English SRP-9001-305 Public | 1.0 |
| Recruitment arrangements (for publication) | K1_ITA Recruitment Procedure Description English SRP-9001-305 Public | 1.0 |
| Subject information and informed consent form (for publication) | BEL Country ICF Procedure English SRP-9001-305 Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_BEL Country ICF Assent Child 13 - maturity Dutch SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_BEL Country ICF Assent Child 13 - maturity English SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_BEL Country ICF Assent Child 13 - maturity French SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_BEL Country ICF Assent Child 7 - 12 years Dutch SRP-9001-305 Public | 3.0 |
| Subject information and informed consent form (for publication) | L1_BEL Country ICF Assent Child 7 - 12 years English SRP-9001-305 Public | 3.0 |
| Subject information and informed consent form (for publication) | L1_BEL Country ICF Assent Child 7 - 12 years French SRP-9001-305 Public | 3.0 |
| Subject information and informed consent form (for publication) | L1_BEL Country ICF Main Dutch SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_BEL Country ICF Main English SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_BEL Country ICF Main French SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_BEL Country ICF Other Pregnant Partner Dutch SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_BEL Country ICF Other Pregnant Partner English SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_BEL Country ICF Other Pregnant Partner French SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_DEU Country ICF Assent Child 13 - 17 years German SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_DEU Country ICF Assent Child 7 - 12 years German SRP-9001-305 Public | 3.0 |
| Subject information and informed consent form (for publication) | L1_DEU Country ICF Main German SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_DEU Country ICF Other Pregnant Partner German SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_DEU Country ICF Procedure English SRP-9001-305 Public | 1 |
| Subject information and informed consent form (for publication) | L1_DEU Country ICF Research German SRP-9001-305 Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_ESP Country ICF Assent Spanish SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ESP Country ICF Main Spanish SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ESP Country ICF Other Pregnant Partner Spanish SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ITA Country ICF Assent 12-17y Italian SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ITA Country ICF Assent 6-11y Italian SRP-9001-305 Public | 3.0 |
| Subject information and informed consent form (for publication) | L1_ITA Country ICF Caregiver Italian SRP-9001-305 Public | 4.1 |
| Subject information and informed consent form (for publication) | L1_ITA Country ICF Main Italian SRP-9001-305 Public | 4.1 |
| Subject information and informed consent form (for publication) | L1_ITA Country ICF Pregnant Partner Italian SRP-9001-305 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ITA Country ICF Research Italian SRP-9001-305 Public | 3.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis Main Dutch SRP-9001-305 Public | 2.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis Main English SRP-9001-305 Public | 2.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis Main French SRP-9001-305 Public | 2.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis Main German SRP-9001-305 Public | 2.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis Main Italian SRP-9001-305 Public | 2.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis Main Spanish SRP-9001-305 Public | 2.0 |
Application history
8 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-11-17 | Spain | Acceptable 2024-03-14
|
2024-03-14 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-05-16 | Spain | Acceptable 2024-07-15
|
2024-07-15 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-09-23 | Acceptable | 2024-11-04 | |
| 4 | SUBSTANTIAL MODIFICATION | SM-3 | 2024-10-07 | Acceptable | 2024-10-17 | |
| 5 | SUBSTANTIAL MODIFICATION | SM-4 | 2024-10-07 | Spain | Acceptable | 2024-11-12 |
| 6 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-03-27 | Spain | Acceptable | 2025-03-27 |
| 7 | SUBSTANTIAL MODIFICATION | SM-5 | 2025-07-09 | Spain | Acceptable 2025-10-01
|
2025-10-02 |
| 8 | SUBSTANTIAL MODIFICATION | SM-7 | 2025-12-19 | Spain | Acceptable 2026-04-13
|
2026-04-14 |