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

Stargardt's Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT01736592
• Other study ID #: LTS13588
• Secondary ID: SG1/002/112012-0
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: December 14, 2012
• Est. completion date: August 29, 2033

Study information

• Verified date: January 2024
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 27
• Est. completion date: August 29, 2033
• Est. primary completion date: August 29, 2033
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Macular Degeneration
• Stargardt Disease
• Stargardt's Disease

Intervention

Drug:
• Long term follow up in all patients who received SAR422459 in previous study TDU13583
Blood draw for the laboratory assessment

Locations

Country	Name	City	State
France	Investigational Site Number : 250001	Paris
United States	Oregon Health and Science University Site Number : 840001	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Sanofi

Countries where clinical trial is conducted

United States,  France, 

Outcome

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