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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-513500-34 | Registry Identifier | CTIS | |
| US1/002/13 | Other Identifier | Sanofi Identifier | |
| 2013-000597-29 | EudraCT Number |
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Primary Objective:
To evaluate the long-term safety and tolerability of SAR421869 in patients with Usher syndrome Type 1B
Secondary Objective:
To assess long-term safety and biological activity of SAR421869
The total duration of study period is up to 15 years.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Long-term follow up | Other | Long-term follow up of patients who received SAR421869 in a previous study TDU13600 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Blood draw for the laboratory assessment | Drug |
|
| 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 (TDU13600) 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 :
Provide signed and dated written informed consent (and if appropriate assent) and any locally required authorization eg, Health Insurance Portability and Accountability Act (HIPAA).
Must have been enrolled in protocol TDU13600. Must have received a subretinal injection of SAR421869
Exclusion criteria:
Did not receive SAR421869 as part of the TDU13600 protocol.
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Sciences & Operations, MD | Sanofi | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Oregon Health and Science University Site Number : 840001 | Portland | Oregon | 97239-3098 | United States | ||
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| Label | URL |
|---|---|
| LTS13619 Plain Language Results Summary | View source |
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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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Measured as change from baseline in function relative to untreated contralateral eye on: BCVA, static perimetry, autofluorescence, optical coherence tomography (OCT) |
| baseline to 15 years |
| Investigational Site Number : 250001 |
| Paris |
| 75012 |
| France |
| ID | Term |
|---|---|
| D052245 | Usher Syndromes |
| ID | Term |
|---|---|
| D054062 | Deaf-Blind Disorders |
| D003638 | Deafness |
| D034381 | Hearing Loss |
| D006311 | Hearing Disorders |
| D004427 | Ear Diseases |
| D010038 | Otorhinolaryngologic Diseases |
| D006319 | Hearing Loss, Sensorineural |
| D012678 | Sensation Disorders |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D001766 | Blindness |
| D014786 | Vision Disorders |
| D012174 | Retinitis Pigmentosa |
| D058499 | Retinal Dystrophies |
| D012162 | Retinal Degeneration |
| D012164 | Retinal Diseases |
| D005128 | Eye Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D015785 | Eye Diseases, Hereditary |
| D030342 | Genetic Diseases, Inborn |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| D001800 | Blood Specimen Collection |
| ID | Term |
|---|---|
| D013048 | Specimen Handling |
| D019411 | Clinical Laboratory Techniques |
| D019937 | Diagnostic Techniques and Procedures |
| D003933 | Diagnosis |
| D011677 | Punctures |
| D013514 | Surgical Procedures, Operative |
| D008919 | Investigative Techniques |
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