Overview
Sponsor-declared trial summary
Congenital, hereditary and neonatal diseases
To evaluate the long-term safety and tolerability of SAR422459 in patients with Stargardt’s macular degeneration.
Key facts
- Sponsor
- Sanofi-Aventis Recherche & Developpement
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Eye Diseases [C11]
- Trial duration
- 30 May 2013 → ongoing
- Decision date (initial)
- 2024-08-13
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Sanofi-Aventis Recherche & Développement
External identifiers
- EU CT number
- 2024-513501-31-00
- EudraCT number
- 2012-001990-95
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy
To evaluate the long-term safety and tolerability of SAR422459 in patients with Stargardt’s macular degeneration.
Secondary objectives 1
- To assess safety and biological activity
Conditions and MedDRA coding
Congenital, hereditary and neonatal diseases
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.1 | PT | 10062766 | Stargardt's disease | 100000004850 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Provide signed and dated written informed consent and any locally required authorization eg, HIPAA
- Must have been enrolled in protocol TDU13583
- Must have received a subretinal injection of SAR422459
- Must have completed protocol TDU13583 to Week 48 or undergone an early discontinuation visit
Exclusion criteria 1
- Did not receive SAR422459 as part of the TDU13583 protocol
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The incidence of Adverse Events
Secondary endpoints 2
- Clinically important changes in ocular safety assessments
- Delay in retinal degeneration
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD11418073 · Product
- Active substance
- Lentiviral Vector Containing the Human ABCA4 Gene
- Substance synonyms
- SAR422459
- Pharmaceutical form
- SUSPENSION FOR INJECTION
- Route of administration
- UNKNOWN USE
- Max daily dose
- 0 DF dosage form
- Max total dose
- 0 DF dosage form
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Not Authorised
- MA holder
- SANOFI AVENTIS RECHERCHE ET DEVELOPPEMENT (SAR)
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/09/720
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Sanofi-Aventis Recherche & Developpement
- Sponsor organisation
- Sanofi-Aventis Recherche & Developpement
- Address
- 13 Quai Jules Guesde
- City
- Vitry Sur Seine
- Postcode
- 94400
- Country
- France
Scientific contact point
- Organisation
- Sanofi-Aventis Recherche & Developpement
- Contact name
- Clinical Sciences and Operations
Public contact point
- Organisation
- Sanofi-Aventis Recherche & Developpement
- Contact name
- Clinical Sciences and Operations
Third parties 2
| Organisation | City, country | Duties |
|---|---|---|
| ESMS Global Limited ORG-100023149
|
London, United Kingdom | Other |
| The Emmes Company LLC ORG-100048299
|
Rockville, United States | Data management |
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ongoing, recruitment ended | 14 | 1 |
| Rest of world
United States
|
— | 13 | — |
Investigational sites
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 |
|---|---|---|---|---|---|
| France | 2013-05-30 | 2013-05-30 | 2019-03-28 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 5 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | d1-rdct-protocol-en-2024-513501-31 | 3 |
| Recruitment arrangements (for publication) | K1-recruitment-arrangements-en-waiver | 1 |
| Subject information and informed consent form (for publication) | L1-sis-icf-addendum-fr | 1 |
| Subject information and informed consent form (for publication) | L1-sis-icf-main-fr | 1 |
| Synopsis of the protocol (for publication) | d1-lay-protocol-synopsis-en-2024-513501-31 | 1 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-07-30 | France | Acceptable 2024-08-09
|
2024-08-13 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2026-04-01 | France | Acceptable 2024-08-09
|
2026-04-01 |