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

Primary Purpose

Stargardt's Disease

Status

Active

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

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

About this trial

This is an interventional other trial for Stargardt's Disease

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Patients must meet ALL of the following criteria:

Provide signed and dated written informed consent and any locally required authorization (e.g., Health Insurance Portability and Accountability Act [HIPAA])
Must have been enrolled in protocol TDU13583 (SG1/001/10)
Must have received a subretinal injection of SAR422459
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.

Sites / Locations

Investigational Site Number 840001
Investigational Site Number 250001

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Long Term Follow up

Arm Description

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

Outcomes

Primary Outcome Measures

The incidence of Adverse Events

The number and percentage of patients with treatment emergent adverse events

Secondary Outcome Measures

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

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)

Full Information

NCT ID

NCT01736592

First Posted

November 26, 2012

Last Updated

June 1, 2023

Sponsor

Sanofi

1. Study Identification

Unique Protocol Identification Number

NCT01736592

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

June 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

December 14, 2012 (Actual)

Primary Completion Date

August 29, 2033 (Anticipated)

Study Completion Date

August 29, 2033 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Sanofi

4. Oversight

Data Monitoring Committee

5. 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

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Stargardt's Disease

7. Study Design

Primary Purpose

Other

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

27 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Long Term Follow up

Arm Type

Other

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

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

Intervention Description

Blood draw for the laboratory assessment

Primary Outcome Measure Information:

Title

The incidence of Adverse Events

Description

The number and percentage of patients with treatment emergent adverse events

Time Frame

15 years

Secondary Outcome Measure Information:

Title

Clinically important changes in ocular safety assessments

Description

Time Frame

baseline to 15 years

Title

Delay in retinal degeneration

Description

Measured as change from baseline in function relative to untreated contralateral eye on: BCVA, static perimetry, microperimetry, autofluorescence, optical coherence tomography (OCT)

Time Frame

baseline to 15 years

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Patients must meet ALL of the following criteria: Provide signed and dated written informed consent and any locally required authorization (e.g., Health Insurance Portability and Accountability Act [HIPAA]) Must have been enrolled in protocol TDU13583 (SG1/001/10) Must have received a subretinal injection of SAR422459 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

David Wilson, MD

Organizational Affiliation

Oregon Health and Science University

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

José-Alain Sahel, MD

Organizational Affiliation

Centre National d'Ophtalmologie des Quinze-Vingts

Official's Role

Principal Investigator

Facility Information:

Facility Name

Investigational Site Number 840001

City

Portland

State/Province

Oregon

ZIP/Postal Code

97239-3098

Country

United States

Facility Name

Investigational Site Number 250001

City

Paris

ZIP/Postal Code

75012

Country

France

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing 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

Learn more about this trial

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

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