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

This study is enrolling participants by invitation only.

Sponsor:

Information provided by (Responsible Party):

Sanofi

ClinicalTrials.gov Identifier:

NCT01736592

First received: November 26, 2012

Last updated: July 11, 2017

Last verified: July 2017

History of Changes

Purpose

The purpose of the study is to examine the long term safety of an experimental gene transfer agent, SAR422459, designed to treat Stargardt Macular Degeneration.

Condition	Intervention	Phase
Stargardt Disease	Drug: SAR422459	Phase 1 Phase 2


Study Type:	Interventional
Study Design:	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

Resource links provided by NLM:

Genetics Home Reference related topics: Stargardt macular degeneration age-related macular degeneration

MedlinePlus related topics: Macular Degeneration

Genetic and Rare Diseases Information Center resources: Stargardt Disease

U.S. FDA Resources

Further study details as provided by Sanofi:

Primary Outcome Measures:

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

Secondary Outcome Measures:

Delay in retinal degeneration [ Time Frame: 15 years ]
The change from baseline function relative to the contralateral eye utilising retinal analytical techniques


Estimated Enrollment:	28
Actual Study Start Date:	December 14, 2012
Estimated Study Completion Date:	March 15, 2034
Estimated Primary Completion Date:	March 15, 2034 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Long Term Follow up Long Term follow up of patients who received SAR422459 in a previous study	Drug: SAR422459 Other Name: Long Term follow up of patients who received SAR422459 in a previous study

Eligibility


Ages Eligible for Study:	18 Years and older (Adult, Senior)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Must have received a subretinal injection of SAR422459.
Must have completed protocol TDU13583 to Week 48 or undergone an early discontinuation visit.

Exclusion Criteria:

Did not receive SAR422459 as part of the TDU13583 protocol

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01736592

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 History of Changes
Other Study ID Numbers:	LTS13588 SG1/002/11
Study First Received:	November 26, 2012
Last Updated:	July 11, 2017

Additional relevant MeSH terms:


Macular Degeneration Retinal Degeneration Retinal Diseases Eye Diseases

ClinicalTrials.gov processed this record on September 25, 2017

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