Clinical trial • Phase IV • Ophthalmology
LENTIVIRAL VECTOR CONTAINING THE HUMAN ABCA4 GENE for Stargardt disease | Stargardt macular degeneration
Phase IV trial of LENTIVIRAL VECTOR CONTAINING THE HUMAN ABCA4 GENE for Stargardt disease | Stargardt macular degeneration.
Overview
- Trial Therapeutic Area
- Ophthalmology
- Trial Disease
- Stargardt disease | Stargardt macular degeneration
- Trial Stage
- Phase IV
- Drug Modality
- Gene therapy
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 30-07-2024
- First CTIS Authorization Date
- 13-08-2024
Trial design
open-label, none/not specified-controlled Phase IV trial across 1 site in France.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 14
Eligibility
Recruits 14 Vulnerable population selected (isVulnerablePopulationSelected = true). Informed consent is required: "Provide signed and dated written informed consent and any locally required authorization eg, HIPAA". Subject information and informed consent forms are listed for publication (L1-sis-icf-main-fr, L1-sis-icf-addendum-fr). No specific assent process for minors is described in the available documents..
- Vulnerable Population
- Vulnerable population selected (isVulnerablePopulationSelected = true). Informed consent is required: "Provide signed and dated written informed consent and any locally required authorization eg, HIPAA". Subject information and informed consent forms are listed for publication (L1-sis-icf-main-fr, L1-sis-icf-addendum-fr). No specific assent process for minors is described in the available documents.
Inclusion criteria
- {"criterion_text":"- Provide signed and dated written informed consent and any locally required authorization eg, HIPAA\n- Must have been enrolled in protocol TDU13583\n- Must have received a subretinal injection of SAR422459\n- Must have completed protocol TDU13583 to Week 48 or undergone an early discontinuation visit"}
Exclusion criteria
- {"criterion_text":"- Did not receive SAR422459 as part of the TDU13583 protocol"}
Endpoints
Primary endpoints
- {"endpoint_text":"- The incidence of Adverse Events","definition_or_measurement_approach":""}
Secondary endpoints
- {"endpoint_text":"- Clinically important changes in ocular safety assessments","definition_or_measurement_approach":""}
- {"endpoint_text":"- Delay in retinal degeneration","definition_or_measurement_approach":""}
Recruitment
- Planned Sample Size
- 14
- Recruitment Window Months
- 242
- Consent Approach
- Participants must provide written informed consent: "Provide signed and dated written informed consent and any locally required authorization eg, HIPAA". Subject information and informed consent forms are available (documents: L1-sis-icf-main-fr, L1-sis-icf-addendum-fr). No age-specific assent details or list of available languages beyond the French consent documents are provided in the available records.
Methods
- Recruitment limited to participants previously enrolled in protocol TDU13583 and who received SAR422459 (i.e., recruitment from prior trial TDU13583).
Geography
- Total Number Of Sites
- 1
- Total Number Of Participants
- 14
France
- Earliest CTIS Part Ii Submission Date
- 27-05-2024
- Latest Decision Or Authorization Date
- 13-08-2024
- Processing Time Days
- 78
- Number Of Sites
- 1
- Number Of Participants
- 14
Sites
- Site Name
- Quinze-Vingts National Ophthalmology Hospital
- Department Name
- Ophtalmology
- Principal Investigator Name
- Jose-Alain SAHEL
- Principal Investigator Email
- jsahel@quinze-vingts.fr
- Contact Person Name
- Jose-Alain SAHEL
- Contact Person Email
- jsahel@quinze-vingts.fr
- Number Of Participants
- 14
Sponsor
Primary sponsor
- Full Name
- Sanofi-Aventis Recherche & Developpement
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- France
Contract research organisations
- Name
- ESMS Global Limited
- Responsibilities
- Centralized 24-Hour Emergency System: eSMS
- Name
- The Emmes Company LLC
Third parties
- {"country":"United Kingdom","full_name":"ESMS Global Limited","duties_or_roles":"Centralized 24-Hour Emergency System: eSMS","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"The Emmes Company LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- SAR422459
- Active Substance
- LENTIVIRAL VECTOR CONTAINING THE HUMAN ABCA4 GENE
- Modality
- Gene therapy
- Routes Of Administration
- Subretinal injection; Suspension for injection
- Route
- Subretinal injection
- Authorisation Status
- prodAuthStatus = 1
- Orphan Designation
- Yes
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