Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration

Study Overview
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Brief Summary
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Primary Objective: To evaluate the long-term safety and tolerability of SAR422459 in patients with Stargardt's Macular Degeneration. Secondary Objective: To assess: Safety Biological activity

Detailed Description
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Patients will be followed for 15 years after completion/early termination from the previous TDU13583 study (NCT01367444). As the initial TDU13583 study is terminated - recruitment to this LTS13588 study is over with 27 patients enrolled.

Official Title
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An Open Label Study to Determine the Long Term Safety, Tolerability and Biological Activity of SAR422459 in Patients With Stargardt's Macular Degeneration

Conditions
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Stargardt's Disease

Intervention / Treatment
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* Drug: Long term follow up in all patients who received SAR422459 in previous study TDU13583


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients must meet ALL of the following criteria: Provide signed and dated written informed consent and any locally required authorization (e.g., Health Insurance Portability and Accountability Act [HIPAA]) Must have been enrolled in protocol TDU13583 (SG1/001/10) Must have received a subretinal injection of SAR422459 Must have completed protocol TDU13583 to Week 48 or undergone an early discontinuation visit. Exclusion Criteria: The following would exclude Patients from participation in the study: 1. Did not receive SAR422459 as part of the TDU13583 protocol.

Ages Eligible for Study
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Minimum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Long Term Follow up<br>Long term follow up in all patients who received SAR422459 in previous study TDU13583 | Drug: Long term follow up in all patients who received SAR422459 in previous study TDU13583<br>* Blood draw for the laboratory assessment<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The incidence of Adverse Events | The number and percentage of patients with treatment emergent adverse events | 15 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinically important changes in ocular safety assessments | From baseline in (TDU13583) study and from the last visit best-corrected visual acuity (BCVA), slit-lamp examination, Fundososcopy, intraocular pressure, laboratory parameters, concomitant medications | baseline to 15 years | 
| Delay in retinal degeneration | Measured as change from baseline in function relative to untreated contralateral eye on: BCVA, static perimetry, microperimetry, autofluorescence, optical coherence tomography (OCT) | baseline to 15 years |