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

• ClinicalTrials.gov Identifier: NCT01736592
• Recruitment Status: Active, not recruiting
• First Posted: November 29, 2012
• Last Update Posted: May 10, 2022
• Sponsor: Sanofi
• Information provided by (Responsible Party): Sanofi

Study Description

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

Condition or disease	Intervention/treatment	Phase
Stargardt's Disease	Drug: Long term follow up in all patients who received SAR422459 in previous study TDU13583	Phase 1; Phase 2

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 27 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Other
• 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
• Actual Study Start Date: December 14, 2012
• Estimated Primary Completion Date: August 2034
• Estimated Study Completion Date: August 2034

Arms and Interventions

Arm	Intervention/treatment
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

Outcome Measures

Primary Outcome Measures :

1. The incidence of Adverse Events [ Time Frame: 15 years ]
   The number and percentage of patients with treatment emergent adverse events

Secondary Outcome Measures :

1. Clinically important changes in ocular safety assessments [ Time Frame: 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
2. Delay in retinal degeneration [ Time Frame: 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)

Eligibility Criteria

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

Criteria

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.

Contacts and Locations

Locations

United States, Oregon

Investigational Site Number 840001

Portland, Oregon, United States, 97239-3098

France

Investigational Site Number 250001

Paris, France, 75012

Sponsors and Collaborators

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

More Information

• Responsible Party: Sanofi
• ClinicalTrials.gov Identifier: NCT01736592
• Other Study ID Numbers: LTS13588; SG1/002/11
• First Posted: November 29, 2012
• Last Update Posted: May 10, 2022
• Last Verified: May 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• 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

Additional relevant MeSH terms:

Macular Degeneration; Stargardt Disease; Retinal Degeneration; Retinal Diseases; Eye Diseases; Eye Diseases, Hereditary; Genetic Diseases, Inborn