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

Phase 2Active Not RecruitingNCT01736592

Sanofi27 enrolled

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

OTHER

Masking

NONE

Enrollment

Conditions

Stargardt's Disease

Interventions

Long term follow up in all patients who received SAR422459 in previous study TDU13583DRUG

Blood draw for the laboratory assessment

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

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. Exclusion Criteria: The following would exclude Patients from participation in the study: 1\. Did not receive SAR422459 as part of the TDU13583 protocol.

Locations (2)

Oregon Health and Science University Site Number : 840001

Portland, Oregon, United States

Investigational Site Number : 250001

Paris, France

Outcomes

Primary Outcomes

The incidence of Adverse Events

The number and percentage of patients with treatment emergent adverse events

Time frame: 15 years

Secondary Outcomes

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

Time frame: 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)

Time frame: baseline to 15 years

Data from ClinicalTrials.gov

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