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| ID | Type | Description | Link |
|---|---|---|---|
| SG1/002/11 | Other Identifier | Sanofi Identifier | |
| 2024-513501-31 | Registry Identifier | CTIS | |
| 2012-001990-95 | EudraCT Number |
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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:
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Long Term Follow up | Other | Long term follow up in all patients who received SAR422459 in previous study TDU13583 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Long term follow up in all patients who received SAR422459 in previous study TDU13583 | Drug | Blood draw for the laboratory assessment |
|
| Measure | Description | Time Frame |
|---|---|---|
| The incidence of Adverse Events | The number and percentage of patients with treatment emergent adverse events | 15 years |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | baseline to 15 years |
| Delay in retinal degeneration |
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Inclusion Criteria:
Patients must meet ALL of the following criteria:
Exclusion Criteria:
The following would exclude Patients from participation in the study:
1. Did not receive SAR422459 as part of the TDU13583 protocol.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Oregon Health and Science University Site Number : 840001 | Portland | Oregon | 97239-3098 | United States | ||
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
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| ID | Term |
|---|---|
| D000080362 | Stargardt Disease |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D008268 | Macular Degeneration |
| D012162 | Retinal Degeneration |
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Measured as change from baseline in function relative to untreated contralateral eye on: BCVA, static perimetry, microperimetry, autofluorescence, optical coherence tomography (OCT) |
| baseline to 15 years |
| Investigational Site Number : 250001 |
| Paris |
| 75012 |
| France |
| D012164 |
| Retinal Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |