NCT01736592

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

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
27

participants targeted

Target at below P25 for phase_2

Timeline
89mo left

Started Dec 2012

Longer than P75 for phase_2

Geographic Reach
2 countries

2 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress65%
Dec 2012Aug 2033

First Submitted

Initial submission to the registry

November 26, 2012

Completed
3 days until next milestone

First Posted

Study publicly available on registry

November 29, 2012

Completed
15 days until next milestone

Study Start

First participant enrolled

December 14, 2012

Completed
20.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 29, 2033

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 29, 2033

Last Updated

June 12, 2025

Status Verified

June 1, 2025

Enrollment Period

20.7 years

First QC Date

November 26, 2012

Last Update Submit

June 9, 2025

Conditions

Stargardt's Disease

Outcome Measures

Primary Outcomes (1)

  • The incidence of Adverse Events

    The number and percentage of patients with treatment emergent adverse events

    15 years

Secondary Outcomes (2)

  • Clinically important changes in ocular safety assessments

    baseline to 15 years

  • Delay in retinal degeneration

    baseline to 15 years

Study Arms (1)

Long Term Follow up

OTHER

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

Interventions

Long term follow up in all patients who received SAR422459 in previous study TDU13583DRUG

Blood draw for the laboratory assessment

Long Term Follow up

Eligibility Criteria

Age6 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • The following would exclude Patients from participation in the study:
  • \. Did not receive SAR422459 as part of the TDU13583 protocol.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Oregon Health and Science University Site Number : 840001

Portland, Oregon, 97239-3098, United States

Location

Investigational Site Number : 250001

Paris, 75012, France

Location

MeSH Terms

Conditions

Stargardt Disease

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesMacular DegenerationRetinal DegenerationRetinal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
OTHER
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 26, 2012

First Posted

November 29, 2012

Study Start

December 14, 2012

Primary Completion (Estimated)

August 29, 2033

Study Completion (Estimated)

August 29, 2033

Last Updated

June 12, 2025

Record last verified: 2025-06

Data Sharing

IPD Sharing
Will share

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

Locations

US(1)FR(1)