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

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

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01736592

Enrollment

Registered

2012-11-29

Start date

2012-12-14

Completion date

2033-08-29

Last updated

2026-08-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Stargardt's Disease

Brief summary

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

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.

Interventions

DRUGLong term follow up in all patients who received SAR422459 in previous study TDU13583

Blood draw for the laboratory assessment

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

OTHER

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

6 Years to No maximum

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
The incidence of Adverse Events	15 years	The number and percentage of patients with treatment emergent adverse events

Secondary

Measure	Time frame	Description
Clinically important changes in ocular safety assessments	baseline to 15 years	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
Delay in retinal degeneration	baseline to 15 years	Measured as change from baseline in function relative to untreated contralateral eye on: BCVA, static perimetry, microperimetry, autofluorescence, optical coherence tomography (OCT)

Countries

France, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Aug 27, 2026