Usher's Syndrome
Conditions
Brief summary
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
Detailed description
The total duration of study period is up to 15 years.
Interventions
Sponsors
Sanofi
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The incidence of adverse events | 15 years | The number and percentage of patients with treatment emergent adverse events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Clinically important changes in ocular safety assessments | baseline to 15 years | 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 |
| Delay in retinal degeneration | baseline to 15 years | Measured as change from baseline in function relative to untreated contralateral eye on: BCVA, static perimetry, autofluorescence, optical coherence tomography (OCT) |
Countries
France, United States
Outcome results
None listed