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

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

Study Overview

• Status: Active, not recruiting
• Conditions: Stargardt's Disease
• Intervention / Treatment: Drug: Long term follow up in all patients who received SAR422459 in previous study TDU13583

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.

• Study Type: Interventional
• Enrollment (Actual): 27
• Phase: Phase 2

Contacts and Locations

Study Locations

• France
  • Paris, France, 75012
    • Investigational Site Number : 250001
• United States
  • Oregon
    • Portland, Oregon, United States, 97239-3098
      • Oregon Health and Science University Site Number : 840001

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Long Term Follow up; 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; Blood draw for the laboratory assessment

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Sanofi
• Investigators: Principal Investigator: David Wilson, MD, Oregon Health and Science University; Principal Investigator: José-Alain Sahel, MD, Centre National d'Ophtalmologie des Quinze-Vingts

Study record dates

Study Major Dates

• Study Start (Actual): December 14, 2012
• Primary Completion (Estimated): August 29, 2033
• Study Completion (Estimated): August 29, 2033

Study Registration Dates

• First Submitted: November 26, 2012
• First Submitted That Met QC Criteria: November 28, 2012
• First Posted (Estimated): November 29, 2012

Study Record Updates

• Last Update Posted (Actual): June 12, 2025
• Last Update Submitted That Met QC Criteria: June 9, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Eye Diseases; Eye Diseases, Hereditary; Retinal Diseases; Retinal Degeneration; Stargardt Disease; Macular Degeneration
• Other Study ID Numbers: LTS13588; SG1/002/11 (Other Identifier: Sanofi Identifier); 2012-001990-95 (EudraCT Number); 2024-513501-31 (Registry Identifier: CTIS)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes