Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Temporarily halted
Participants planned
148
Countries
6
Sites
19
Duchenne Muscular Dystrophy
Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72
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
- 28 Mar 2024 → ongoing
- Decision date (initial)
- 2024-09-12
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Sarepta Therapeutics, Inc.
External identifiers
- EU CT number
- 2024-512626-28-00
- EudraCT number
- 2020-002372-13
- ClinicalTrials.gov
- NCT05881408
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy, Efficacy, Safety
Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72
Secondary objectives 8
- Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72
- Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72
- Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
- Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72
- Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE), Baseline up to Week 124
- Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72
- Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72
- Part 1: Change From Baseline in PUL (Version 2.0) Middle Domain Score at Week 72
Conditions and MedDRA coding
Duchenne Muscular Dystrophy
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.1 | PT | 10052655 | Duchenne muscular dystrophy gene carrier | 100000004850 |
| 20.0 | PT | 10013801 | Duchenne muscular dystrophy | 100000004850 |
Regulatory references
- Scientific advice from competent authorities
- Federal Agency For Medicines And Health Products
- EMA paediatric investigation plan (PIP)
- EMEA-002677-PIP01-19
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 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) | |
| 2023-505043-39-00 | A Phase 3, Multinational, Long-Term Follow-Up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study | Sarepta Therapeutics Inc. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 6
- Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing. A pathogenic frameshift mutation or premature stop codon in the DMD gene, except for any deletion in exons 1 to 17 and/or exons 59 to 71.
- Cohort 1 only: Non-ambulatory per protocol specified criteria.
- Cohort 2 only: Ambulatory per protocol specified criteria and ≥8 to <18 years of age at the time of Screening.
- Ability to cooperate with motor assessment testing.
- Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
- Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements.
Exclusion criteria 4
- Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
- Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
- Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.
- Other inclusion or exclusion criteria could apply.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72
Secondary endpoints 8
- Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72
- Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72
- Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
- Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72
- Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)
- Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72
- Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72
- Part 1: Change From Baseline in PUL (Version 2.0) Middle Domain Score at Week 72.
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
Placebo 1
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
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, Code 14, Other, Interactive response technologies (IRT), Laboratory analysis, Code 5, Data management, E-data capture, Code 8 |
Locations
6 EU/EEA countries · 19 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Belgium | Temporarily halted | 10 | 2 |
| France | Suspended | 10 | 3 |
| Germany | Suspended | 15 | 5 |
| Italy | Temporarily halted | 20 | 4 |
| Spain | Temporarily halted | 25 | 3 |
| Sweden | Temporarily halted | 15 | 2 |
| Rest of world
Korea, Republic of, Israel, Hong Kong, United States, Australia, Taiwan, Japan, Canada, United Kingdom
|
— | 53 | — |
Investigational sites
Centre Hospitalier Regional De La Citadelle
446: Laboratoire, Boulevard Du Douzieme De Ligne 1, 4000, Liege
Universitair Ziekenhuis Gent
406: Neuromuscular Reference Centre (NMRC), Corneel Heymanslaan 10, 9000, Gent
Hopital Necker Enfants Malades
service de Neuropédiatrie, 149 Rue De Sevres, 75015, Paris
Hopital Des Enfants
service de Neuropédiatrie, 330 Avenue De Grande Bretagne, 31059, Toulouse Cedex 9
Hospices Civils De Lyon
service de Neuropédiatrie, 59 Boulevard Pinel, 69500, Bron
University Medical Center Hamburg-Eppendorf
462: Nephrology and Rheumatology, Martinistrasse 52, Eppendorf, Hamburg
Universitaetsklinikum Essen AöR
409: Klinik für Kinderheilkunde I, Hufelandstrasse 55, Holsterhausen, Essen
Klinikum der Universitaet Muenchen AöR
412: NAP, Lindwurmstrasse 4, Ludwigsvorstadt-Isarvorstadt, Munich
Universitaetsklinikum Heidelberg AöR
470: Zentrum für Kinder und Jugendmedizin, Im Neuenheimer Feld 430, Neuenheim, Heidelberg
Charite Universitaetsmedizin Berlin KöR
448: Department of Neuropaediatrics and Center for Chronically Sick Children, Augustenburger Platz 1, Wedding, Berlin
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
UOC Neurologia, Via Francesco Sforza 28, 20122, Milan
IRCCS Istituto Giannina Gaslini
Centro di Miologia Traslazionale e Sperimentale, Via Gerolamo Gaslini 5, 16147, Genoa
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
U.O.C. Neuropsichiatria Infantile, Largo Francesco Vito 1, 00168, Rome
IRCCS Foundation Istituto Neurologico Carlo Besta
S.C. Neuropsichiatria Infantile 2 - Epilettologia e Neurologia dello Sviluppo (NDS), Via Giovanni Celoria 11, 20133, Milan
Hospital Sant Joan De Deu Barcelona
Neuropediatría, Passeig De Sant Joan De Deu 2, 08950, Esplugues De Llobregat
Hospital Universitario Y Politecnico La Fe
Neurología, Avenida De Fernando Abril Martorell 106, 46026, Valencia
Hospital Universitario 12 De Octubre
Neurología /Sección de Neurología Pediátrica, Avenida De Cordoba Sn, 28041, Madrid
Karolinska University Hospital
NAP, Eugeniavagen 3, 171 64, Solna
Queen Silvia Childrens Hospital - Sahlgrenska University Hospital - Vaestra Goetalandsregionen
NAP, Behandlingsvagen 7, Harlanda, Gothenburg
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 | 2024-03-28 | 2024-04-09 | 2025-03-31 | ||
| France | |||||
| Germany | 2024-09-09 | 2024-10-08 | |||
| Italy | 2024-03-01 | 2024-03-05 | 2025-03-31 | ||
| Spain | 2024-02-12 | 2024-02-19 | 2025-03-31 | ||
| Sweden | 2024-10-16 | 2024-11-04 | 2025-03-31 |
Oversight and notifications
Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77
Temporary halts 6 · Art. 38 CTR
Temporary halt TH-77173
- Halt date
- 2025-03-31
- Member states concerned
- Italy
- Publication date
- 2025-03-31
- Reason
- Safety related (clinical or pre-clinical results)
- Explanation
- In response to the post marketing Acute liver Failure (ALF) safety event communicated through a Dear Investigator Letter (DIL) enrollment and dosing is temporary halted at the request of EU regulators.
- Follow-up measures
- The Sponsor will continue patient safety monitoring for already enrolled patients and continue recording data.
- Benefit-risk balance changed
- Yes
- Treatment stopped
- Yes
Temporary halt TH-77174
- Halt date
- 2025-03-31
- Member states concerned
- Belgium
- Publication date
- 2025-03-31
- Reason
- Safety related (clinical or pre-clinical results)
- Explanation
- In response to the post marketing Acute liver Failure (ALF) safety event communicated through a Dear Investigator Letter (DIL) enrollment and dosing is temporary halted at the request of EU regulators
- Follow-up measures
- The Sponsor will continue patient safety monitoring for already enrolled patients and continue recording data.
- Benefit-risk balance changed
- Yes
- Treatment stopped
- Yes
Temporary halt TH-51317
- Halt date
- 2024-08-23
- Planned restart
- 2024-11-30
- Member states concerned
- Italy
- Publication date
- 2024-10-14
- Reason
- Sponsor decision
- Explanation
- The Sponsor, Sarepta Therapeutics, temporarily paused recruitment for the ENVISION study in Italy and Spain on 23Aug2024 due to a pause in AAVrh74 Antibody ELISA sample testing by their Diagnostic Device Partner, Quest Diagnostics, in their clinical performance study (DM-SC-IVD1-PRE 44/2023, CIV-ID: CIV-SE-23-02-042372, no. 44/2023). This pause is not related to changes in the SRP-9001 benefit-risk profile or safety concerns with SRP-9001 or with the Quest ELISA assay.
Quest Diagnostics has submitted substantial modifications to Quest’s clinical performance study to the relevant Competent Authorities and Ethics Committees in these EU Countries to inform of an increase in the number of samples Quest plans to test using the AAVrh74 Antibody ELISA. This increase in the number of samples is intended to align with the current screening and enrollment estimates Sarepta has for the SRP-9001-303 ENVISION study in each member state.
Sarepta would like to clarify that the total sample size of 148 for the ENVISION study remains unchanged.
SRP-9001 is a one-time infusion. To date, 57 subjects have been infused globally, including 12 subjects in Italy and 15 subjects in Spain. Recruitment is ongoing in other countries including Belgium, Japan, the United Kingdom, Australia, Germany, and Taiwan. - Follow-up measures
- Not applicable as the pause in recruitment is not due to any safety concerns or changes to the benefit-risk profile.
- Benefit-risk balance changed
- No
- Treatment stopped
- Yes
Temporary halt TH-77171
- Halt date
- 2025-03-31
- Member states concerned
- Sweden
- Publication date
- 2025-03-31
- Reason
- Safety related (clinical or pre-clinical results)
- Explanation
- In response to the post marketing Acute liver Failure (ALF) safety event communicated through a Dear Investigator Letter (DIL) enrollment and dosing is temporary halted at the request of EU regulators.
- Follow-up measures
- The Sponsor will continue patient safety monitoring for already enrolled patients and continue recording data.
- Benefit-risk balance changed
- Yes
- Treatment stopped
- Yes
Temporary halt TH-77172
- Halt date
- 2025-03-31
- Member states concerned
- Spain
- Publication date
- 2025-03-31
- Reason
- Safety related (clinical or pre-clinical results)
- Explanation
- In response to the post marketing Acute liver Failure (ALF) safety event communicated through a Dear Investigator Letter (DIL) enrollment and dosing is temporary halted at the request of EU regulators.
- Follow-up measures
- The Sponsor will continue patient safety monitoring for already enrolled patients and continue recording data.
- Benefit-risk balance changed
- Yes
- Treatment stopped
- Yes
Temporary halt TH-52128
- Halt date
- 2024-08-23
- Planned restart
- 2024-11-30
- Member states concerned
- Spain
- Publication date
- 2024-10-18
- Reason
- Sponsor decision
- Explanation
- The Sponsor, Sarepta Therapeutics, temporarily paused recruitment for the ENVISION study in Italy and Spain on 23Aug2024 due to a pause in AAVrh74 Antibody ELISA sample testing by their Diagnostic Device Partner, Quest Diagnostics, in their clinical performance study (DM-SC-IVD1-PRE 44/2023, CIV-ID: CIV-SE-23-02-042372, no. 44/2023). This pause is not related to changes in the SRP-9001 benefit-risk profile or safety concerns with SRP-9001 or with the Quest ELISA assay.
Quest Diagnostics has submitted substantial modifications to Quest’s clinical performance study to the relevant Competent Authorities and Ethics Committees in these EU Countries to inform of an increase in the number of samples Quest plans to test using the AAVrh74 Antibody ELISA. This increase in the number of samples is intended to align with the current screening and enrollment estimates Sarepta has for the SRP-9001-303 ENVISION study in each member state.
Sarepta would like to clarify that the total sample size of 148 for the ENVISION study remains unchanged.
SRP-9001 is a one-time infusion. To date, 57 subjects have been infused globally, including 12 subjects in Italy and 15 subjects in Spain. Recruitment is ongoing in other countries including Belgium, Japan, the United Kingdom, Australia, Germany, and Taiwan. - Follow-up measures
- Not applicable as the pause in recruitment is not due to any safety concerns or changes to the benefit-risk profile.
- Benefit-risk balance changed
- No
- Treatment stopped
- Yes
Corrective measures 2 · Art. 77 CTR
Corrective measure CM-DE-0001
- Member state
- Germany
- Publication date
- 2025-03-31
- Type
- 1
- Reason
- 5, 7
- Immediate action required
- Yes
Corrective measure CM-FR-0001
- Member state
- France
- Publication date
- 2025-04-02
- Type
- 1
- Reason
- 5
- Immediate action required
- Yes
- Justification
- After receipt of the Dear Investigator Letter: “Delandistrogene moxeparvovec: fatal case of acute liver failure and intracranial hemorrhage” dated 18 Mar 2025, we ask the Sponsor to immediately put on hold enrollment in France, until analysis of the fatal case has been finalised by the RMSs.
The temporay halt was already notified in the other european countries concerned, except France as the clinical trial had not started. The sponsor should confirm that the enrollment, is put on hold in France.
Furthermore, the Sponsor has to notify this fatal case through the EU portal without undue delay.
Indeed and according to the article 53 of the REGULATION (EU) No 536/2014 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 16 April 2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC, the sponsor shall notify the Member States concerned through the EU portal of all unexpected events which affect the benefit-risk balance of the clinical trial, but are not suspected unexpected serious adverse reactions as referred to in Article 42. That notification shall be made without undue delay but no later than 15 days from the date the sponsor became aware of this event.
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 173 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_ English_SRP-9001-303_Public | 3.0 |
| Protocol (for publication) | D4_Subject Questionaries_Transparency Placeholder SRP-9001-303 | NA |
| Recruitment arrangements (for publication) | K1_Recruitment Patient Information Letter Italian Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment and Informed Procedure French, English Public | 1.2 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangement Dear Patient Letter Spanish Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangement Other Study Information Sheet Spanish Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangement Other Visit Preparation Guide Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangement Visit Preparation Guide Swedish Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Dear Patient Letter Dutch Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Dear Patient Letter English Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements Dear Patient Letter French Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Dear Patient Letter French Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Disease Fact Sheet French Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements Fact Sheet English Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements Fact Sheet Italian Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Other Disease Fact Sheet Dutch Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Other Disease Fact Sheet English Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Other Information Sheet Dutch Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Other Information Sheet English Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Other Information Sheet French Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Other Preparation Guide Dutch Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Other Preparation Guide English Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Other Preparation Guide French Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Other Study Brochure Dutch Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Other Study Brochure English Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Other Study Brochure French Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Other Study Guide Dutch Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Other Study Guide English Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements Other Study Guide French Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements Participant Fact Sheet French Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements Participant Study Guide French Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements Participant Study Guide Italian Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements Patient Information Letter English Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements Study Information Sheet English Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements Study Information Sheet Italian Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements Visit Participation Guide English Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangment Participant Fact Sheet Spanish Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangments Visit Guide French Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangments Visit preparation Guide Italian Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Brochure Italian Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Brochure Swedish Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Disease Fact Sheet German Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Flyer Study Information Sheet French Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Master Participant Study Guide English Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Other Participant study guide German Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Other Participant Study Guide Spanish Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Other Study Guide Swedish Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Participant Fact Sheet Swedish Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Patient Information Letter German Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Patient Information Letter Swedish Public | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Procedure Description and ICF procedure English Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Procedure Description English Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Procedure English Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Study Information Sheet German Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Study Information Sheet Swedish Public | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Visit Preparation Guide German Public | 1.0 |
| Recruitment arrangements (for publication) | K2_Recruitment Arrangments Study Brochure English Public | 2.0 |
| Recruitment arrangements (for publication) | K2_Recruitment Brochure English Public | 2.0 |
| Recruitment arrangements (for publication) | K2_Recruitment Brochure French Public | 2.0 |
| Recruitment arrangements (for publication) | K2_Recruitment Brochure German Public | 2.0 |
| Recruitment arrangements (for publication) | K2_Recruitment Brochure Spanish Public | 2.0 |
| Recruitment arrangements (for publication) | K2_Recruitment Dear Patient Letter English Public | 2.0 |
| Recruitment arrangements (for publication) | K2_Recruitment Disease Fact Sheet English Public | 1.0 |
| Recruitment arrangements (for publication) | K2_Recruitment Master Participant Study Guide English Public | 2.0 |
| Recruitment arrangements (for publication) | K2_Recruitment Procedure Description English Public | 1.0 |
| Recruitment arrangements (for publication) | K2_Recruitment Procedure Description Swedish Public | 1.0 |
| Recruitment arrangements (for publication) | K2_Recruitment Study information sheet English Public | 1.0 |
| Recruitment arrangements (for publication) | K2_Recrutiment Visit preparation Guide English Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_ ICF Optional Adult Legal Guard Swedish Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_ ICF Other Pregnant Partner English Public | 1.1 |
| Subject information and informed consent form (for publication) | L1_ ICF Research German Public | 1.1 |
| Subject information and informed consent form (for publication) | L1_BEL Country ICF Procedure Sponsor Statement English SRP-9001-303 Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_Country ICF Assent Ages 8-12 French Public | 3.1 |
| Subject information and informed consent form (for publication) | L1_Country ICF Assent 13y English Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_Country ICF Assent 8-12y English Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_Country ICF Assent Ages 13-17 French Public | 3.1 |
| Subject information and informed consent form (for publication) | L1_Country ICF Assent Child 8-12y Italian Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_Country ICF Assent Child 13-17 English Public | 6.0 |
| Subject information and informed consent form (for publication) | L1_Country ICF Main Adult English Public | 3.0 |
| Subject information and informed consent form (for publication) | L1_Country ICF Main Adult French Public | 3.2 |
| Subject information and informed consent form (for publication) | L1_Country ICF Main Adult Italian Public | 7.0 |
| Subject information and informed consent form (for publication) | L1_Country ICF Main atteigning 18 years French Public | 3.2 |
| Subject information and informed consent form (for publication) | L1_Country ICF Main Parent Legal Tutor English Public | 3.0 |
| Subject information and informed consent form (for publication) | L1_Country ICF Main parents French Public | 3.2 |
| Subject information and informed consent form (for publication) | L1_Country ICF Main Study Parent English | 7.0 |
| Subject information and informed consent form (for publication) | L1_Country ICF Optional Research English Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_Country ICF Other Parents-legal guardian English Public | 7.0 |
| Subject information and informed consent form (for publication) | L1_Country ICF Pregnant Partner English Public | 1.1 |
| Subject information and informed consent form (for publication) | L1_Country ICF Research English Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_Country ICF Research Italian Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_Country ICF Travel expenses Reimbursement English Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_Country Main ICF Adult English Public | 7.0 |
| Subject information and informed consent form (for publication) | L1_ICF Assent 13 - AOM maturity German Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Assent 15-17 SRP-9001-303 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Assent 16-18 years Dutch Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_ICF Assent 16-18 years_ French Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_ICF Assent 8 - 12 German Public | 3.1 |
| Subject information and informed consent form (for publication) | L1_ICF Assent 8-12 years_ English Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Assent 8-14 Swedish Public | 3.0 |
| Subject information and informed consent form (for publication) | L1_ICF Assent Child 12-17 Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Assent Child 13 - AOM English Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Assent Child 13-15 years French Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Assent Child 13-15 years_Dutch Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Assent Child 13-15 years_English Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Assent Child 16-18 years English Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_ICF Assent Child 8-12 years English Public | 3.1 |
| Subject information and informed consent form (for publication) | L1_ICF Assent Child 8-12 years_ French Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Assent Child 8-12 years_Dutch Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF CI SRP-9001-303 Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_ICF Main 18 or older Swedish Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Main Adult English | 7.0 |
| Subject information and informed consent form (for publication) | L1_ICF Main Adult Spanish Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Main Appendix 1 Swedish Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Main English Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Main German Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Main Legal Guard Swedish Public | 4.0 |
| Subject information and informed consent form (for publication) | L1_ICF Main Parental Dutch Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_ICF Main Parental English Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_ICF Main Parental French Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_ICF Main_ French Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_ICF Main_Dutch Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_ICF Main_English Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_ICF Optional 15-17 Swedish Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_ICF Optional Research English Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF Optional Research German Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_ICF Optional Research Italian Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_ICF Optional Research SRP-9001-303 Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_ICF Optional Research_ Dutch Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_ICF Optional Research_ French Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_ICF Optional Research_English Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_ICF Other Child Minors 12-17 Spanish Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF Other French Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF Other Pregnant Partner English Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF Other Pregnant Partner_Dutch Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF Other Pregnant Partner_French Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF Other Public Dutch | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF Other Travel expenses_SRP-9001-303 Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF Other_English Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF Pregnant Other_SRP-9001-303 Public | 1.1 |
| Subject information and informed consent form (for publication) | L1_ICF Pregnant Partner ICF SRP-9001-303 Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF Programme SRP-9001-303 Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_ICF Research 15-17 Swedish Public | 3.0 |
| Subject information and informed consent form (for publication) | L1_ICF Research Legal Guard Swedish Public | 3.0 |
| Subject information and informed consent form (for publication) | L1_ICF Sub Study Adult - Parents Biopsy Spanish Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF Sub Study Adult Parents Spanish Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_ICF Sub Study Child Biopsy12-17 Spanish Public | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF Subject Materials Program English Public | 1.0 |
| Subject information and informed consent form (for publication) | L1-Country ICF Assent 13-17y Italian Public | 6.0 |
| Subject information and informed consent form (for publication) | L2_Country ICF Main Parent legal tutor ICF Italian Public | 7.0 |
| Subject information and informed consent form (for publication) | L2_ICF Bilingual Public | 2.0 |
| Subject information and informed consent form (for publication) | L2_ICF English Public | 2.0 |
| Subject information and informed consent form (for publication) | L2_ICF English Public | 2.0 |
| Subject information and informed consent form (for publication) | L2_ICF German Public | 2.0 |
| Subject information and informed consent form (for publication) | L2_ICF Other German Public | 1.1 |
| Subject information and informed consent form (for publication) | L2_ICF Other pregnant partner SRP-9001-303 Public | 1.1 |
| Subject information and informed consent form (for publication) | L2_ICF Other Pregnant Partner SRP-9001-303 Public | 1.1 |
| Subject information and informed consent form (for publication) | L2_ICF Other Pregnant Partner Swedish Public | 1.0 |
| Subject information and informed consent form (for publication) | L2_ICF Other Program English Public | 1.1 |
| Subject information and informed consent form (for publication) | L2_ICF Other SRP-9001-303 Public | 1.0 |
| Subject information and informed consent form (for publication) | L2_ICF Other SRP-9001-303 Public | 1.0 |
| Subject information and informed consent form (for publication) | L2_ICF Spanish Public | 2.0 |
| Subject information and informed consent form (for publication) | L2_ICF Swedish Public | 2.0 |
| Subject information and informed consent form (for publication) | L2_Subject Materials Other Dutch Public | 2.0 |
| Subject information and informed consent form (for publication) | L2_Subject Materials Other French Public | 2.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis_Dutch_SRP-9001-303_Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis_English_SRP-9001-303_Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis_French_BE_SRP-9001-303_Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis_French_FR_SRP-9001-303_Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis_German_BE_SRP-9001-303_Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis_German_DE_SRP-9001-303_Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis_Italian _SRP-9001-303_Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis_Spanish_SRP-9001-303_Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis_Swedish_SRP-9001-303_Public | 1.0 |
Application history
3 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-08-09 | Belgium | Acceptable with conditions 2024-09-12
|
2024-09-12 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-12-18 | Belgium | Acceptable with conditions 2025-04-29
|
2025-04-30 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2025-05-19 | Belgium | Acceptable with conditions 2025-08-11
|
2025-08-11 |