Phase I/IIA Study of SAR422459 in Participants With Stargardt's Macular Degeneration
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ClinicalTrials.gov Identifier: NCT01367444 |
Recruitment Status :
Terminated
(Study stopped not for safety reasons. Due to review of clinical development plans and priorities, Sponsor decided to stop development of the product.)
First Posted : June 7, 2011
Results First Posted : June 5, 2020
Last Update Posted : April 14, 2022
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Tracking Information | |||||||
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First Submitted Date ICMJE | June 3, 2011 | ||||||
First Posted Date ICMJE | June 7, 2011 | ||||||
Results First Submitted Date ICMJE | May 19, 2020 | ||||||
Results First Posted Date ICMJE | June 5, 2020 | ||||||
Last Update Posted Date | April 14, 2022 | ||||||
Actual Study Start Date ICMJE | June 8, 2011 | ||||||
Actual Primary Completion Date | August 16, 2019 (Final data collection date for primary outcome measure) | ||||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
The incidence of adverse events [ Time Frame: 48 weeks ] The number and percentage of patients with treatment emergent adverse events.
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Change History | |||||||
Current Secondary Outcome Measures ICMJE | Not Provided | ||||||
Original Secondary Outcome Measures ICMJE |
Delay in retinal degeneration [ Time Frame: 48 weeks ] Changes from baseline function relative to the contralateral eye utilising retinal analytical techniques.
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Current Other Pre-specified Outcome Measures | Not Provided | ||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||
Descriptive Information | |||||||
Brief Title ICMJE | Phase I/IIA Study of SAR422459 in Participants With Stargardt's Macular Degeneration | ||||||
Official Title ICMJE | A Phase I/IIA Dose Escalation Safety Study of Subretinally Injected SAR422459, Administered to Patients With Stargardt's Macular Degeneration | ||||||
Brief Summary | Primary Objective: To assess the safety and tolerability of ascending doses of SAR422459 in participants with Stargardt's Macular Degeneration (SMD). Secondary Objective: To evaluate for possible biological activity of SAR422459. |
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Detailed Description | The total duration per participant was up to 52 weeks, which included 4 week screening period and 48 weeks study period. At the end of the study, the participants were invited to enter in an open-label safety study (LTS13588-NCT01736592) for long-term follow-up visits including ophthalmological examinations and recording of adverse events (AEs) for up to 15 years. |
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Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Phase 1 Phase 2 |
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Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Stargardt's Disease | ||||||
Intervention ICMJE | Drug: SAR422459
Pharmaceutical form: sterile solution, 100 microliters (μL) aliquots in 0.3 milliliter (mL) type I borosilicate glass 'V' vials with a butyl stopper and aluminum crimp seal. Route of administration: subretinal injection |
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Study Arms ICMJE |
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||
Recruitment Status ICMJE | Terminated | ||||||
Actual Enrollment ICMJE |
27 | ||||||
Original Estimated Enrollment ICMJE |
28 | ||||||
Actual Study Completion Date ICMJE | August 16, 2019 | ||||||
Actual Primary Completion Date | August 16, 2019 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Specific Inclusion Criteria Participant Group A:
Specific Inclusion Criteria Participant Group B:
Specific Inclusion Criteria Participant Group C:
Specific Inclusion Criteria Participant Group D:
Specific inclusion criteria Participant Group E:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 6 Years and older (Child, Adult, Older Adult) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||
Listed Location Countries ICMJE | France, United States | ||||||
Removed Location Countries | |||||||
Administrative Information | |||||||
NCT Number ICMJE | NCT01367444 | ||||||
Other Study ID Numbers ICMJE | TDU13583 SG1/001/10 |
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Has Data Monitoring Committee | Yes | ||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Sanofi | ||||||
Original Responsible Party | Dr Graham Price, Oxford BioMedica (UK) Ltd | ||||||
Current Study Sponsor ICMJE | Sanofi | ||||||
Original Study Sponsor ICMJE | Oxford BioMedica | ||||||
Collaborators ICMJE | Not Provided | ||||||
Investigators ICMJE |
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PRS Account | Sanofi | ||||||
Verification Date | March 2022 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |