Clinical trial • Phase IV • Rare Disease|Ophthalmology
LENTIVIRAL VECTOR DERIVED FROM THE EQUINE INFECTIOUS ANAEMIA VIRUS CONTAINING THE HUMAN MYO7A GENE for Usher syndrome type 1B
Phase IV trial of LENTIVIRAL VECTOR DERIVED FROM THE EQUINE INFECTIOUS ANAEMIA VIRUS CONTAINING THE HUMAN MYO7A GENE for Usher syndrome type 1B.
Overview
- Trial Therapeutic Area
- Rare Disease|Ophthalmology
- Trial Disease
- Usher syndrome type 1B
- Trial Stage
- Phase IV
- Drug Modality
- Gene therapy
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 30-07-2024
- First CTIS Authorization Date
- 12-09-2024
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
- 5
Eligibility
Recruits 5 Vulnerable population selected. Consent/assent handling: "Provide signed and dated written informed consent (and if appropriate assent) and any locally required authorization eg, Health Insurance Portability and Accountability Act (HIPAA)." Informed consent documents available in the trial dossier (e.g. L1-sis-icf-main-fr, L1-sis-icf-addendum-fr)..
- Vulnerable Population
- Vulnerable population selected. Consent/assent handling: "Provide signed and dated written informed consent (and if appropriate assent) and any locally required authorization eg, Health Insurance Portability and Accountability Act (HIPAA)." Informed consent documents available in the trial dossier (e.g. L1-sis-icf-main-fr, L1-sis-icf-addendum-fr).
Inclusion criteria
- {"criterion_text":"- Provide signed and dated written informed consent (and if appropriate assent) and any locally required authorization eg, Health Insurance Portability and Accountability Act (HIPAA).\n- Must have been enrolled in protocol TDU13600.\n- Must have received a subretinal injection of SAR421869"}
Exclusion criteria
- {"criterion_text":"- Did not receive SAR421869 as part of the TDU13600 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
- 5
- Recruitment Window Months
- 217
- Consent Approach
- "Provide signed and dated written informed consent (and if appropriate assent) and any locally required authorization eg, Health Insurance Portability and Accountability Act (HIPAA)." Subject information and informed consent form documents in the dossier include L1-sis-icf-main-fr and L1-sis-icf-addendum-fr (French).
Geography
- Total Number Of Sites
- 1
- Total Number Of Participants
- 4
France
- Earliest CTIS Part Ii Submission Date
- 14-08-2024
- Latest Decision Or Authorization Date
- 12-09-2024
- Processing Time Days
- 29
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Quinze-Vingts National Ophthalmology Hospital
- Department Name
- Ophtalomogy
- 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
- 4
Sponsor
Primary sponsor
- Full Name
- Sanofi-Aventis Recherche & Developpement
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- France
Contract research organisations
- Name
- The Emmes Company LLC
- Responsibilities
- sponsorDuties code 6; contact twiens@emmes.com
- Name
- ESMS Global Limited
- Responsibilities
- Centralized 24-Hour Emergency System: eSMS; contact rita.fitzpatrick@esmsglobal.com
Third parties
- {"country":"United States","full_name":"The Emmes Company LLC","duties_or_roles":"sponsorDuties code 6","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"ESMS Global Limited","duties_or_roles":"Centralized 24-Hour Emergency System: eSMS","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- SAR421869
- Active Substance
- LENTIVIRAL VECTOR DERIVED FROM THE EQUINE INFECTIOUS ANAEMIA VIRUS CONTAINING THE HUMAN MYO7A GENE
- Modality
- Gene therapy
- Routes Of Administration
- Subretinal injection (suspension for injection)
- Route
- Subretinal injection
- Orphan Designation
- Yes
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