Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT01736592 |
Recruitment Status :
Active, not recruiting
First Posted : November 29, 2012
Last Update Posted : May 10, 2022
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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 |
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Stargardt's Disease | Drug: Long term follow up in all patients who received SAR422459 in previous study TDU13583 | Phase 1 Phase 2 |
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 |

Arm | Intervention/treatment |
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Long Term Follow up
Long term follow up in all patients who received SAR422459 in previous study TDU13583
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Drug: Long term follow up in all patients who received SAR422459 in previous study TDU13583
Blood draw for the laboratory assessment |
- The incidence of Adverse Events [ Time Frame: 15 years ]The number and percentage of patients with treatment emergent adverse events
- 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
- 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)

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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01736592
United States, Oregon | |
Investigational Site Number 840001 | |
Portland, Oregon, United States, 97239-3098 | |
France | |
Investigational Site Number 250001 | |
Paris, France, 75012 |
Principal Investigator: | David Wilson, MD | Oregon Health and Science University | |
Principal Investigator: | José-Alain Sahel, MD | Centre National d'Ophtalmologie des Quinze-Vingts |
Responsible Party: | Sanofi |
ClinicalTrials.gov Identifier: | NCT01736592 |
Other Study ID Numbers: |
LTS13588 SG1/002/11 |
First Posted: | November 29, 2012 Key Record Dates |
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 |
Macular Degeneration Stargardt Disease Retinal Degeneration Retinal Diseases |
Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn |