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

Phase 1

Conditions: Stargardt Macular Dystrophy, also known as fundus flavimaculatus or Stargardt disease.
MedDRA version: 20.1Level: PTClassification code 10062766Term: Stargardt's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Eye Diseases [C11]

Registration Number: EUCTR2012-001990-95-FR

Lead Sponsor: sanofi-aventis recherche et développement

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 27

Inclusion Criteria

Patients must meet ALL of the following criteria:
1) Provide signed and dated written informed consent and any locally required authorization (e.g., Health Insurance Portability and Accountability Act [HIPAA]).
2) Must have been enrolled in protocol TDU13583 (SG1/001/10).
3) Must have received a subretinal injection of SAR422459
4) Must have completed protocol TDU13583 to Week 48 or undergone an early discontinuation visit.
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 26
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

The following would exclude patients from participation in the study:
1) Did not receive SAR422459 as part of the TDU13583 protocol

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the long term safety and tolerability of SAR422459 in patients with Stargardt’s macular degeneration.;Secondary Objective: To Assess<br>• Safety<br>• Biological Activity;Primary end point(s): The incidence of Adverse Events: The number and percentage of patients with treatment emergent adverse events;Timepoint(s) of evaluation of this end point: 5 years

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Clinical 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, lab parameters, concomitant meds<br>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);Timepoint(s) of evaluation of this end point: 1, 2 : Baseline to 15 years