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

Phase 1

Active, not recruiting

• Conditions: Stargardt's Disease
• Interventions: Drug: Long term follow up in all patients who received SAR422459 in previous study TDU13583

• Registration Number: NCT01736592
• Lead Sponsor: Sanofi

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.

Study Timeline

• Study First Submitted: 2012-11-26
• Study First Submitted QC: 2012-11-28
• Study First Posted: 2012-11-29
• Study Start: 2012-12-14
• Status Verified: 2024-01
• Last Update Submitted: 2024-01-09
• Last Update Posted: 2024-01-10
• Primary Completion: 2033-08-29
• Study Completion: 2033-08-29

Recruitment & Eligibility

• Status: ACTIVE_NOT_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.

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
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Long Term Follow up	Long term follow up in all patients who received SAR422459 in previous study TDU13583	Long term follow up in all patients who received SAR422459 in previous study TDU13583

Primary Outcome Measures

Name	Time	Method
The incidence of Adverse Events	15 years	The number and percentage of patients with treatment emergent adverse events

Secondary Outcome Measures

Name	Time	Method
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)

Trial Locations

Locations (2)

Investigational Site Number : 250001; 🇫🇷 Paris, France

Oregon Health and Science University Site Number : 840001; 🇺🇸 Portland, Oregon, United States