A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B
- Conditions
- Usher's Syndrome
- Interventions
- Drug: Blood draw for the laboratory assessment
- Registration Number
- NCT02065011
- Lead Sponsor
- Sanofi
- 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 9
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Long-term follow up Blood draw for the laboratory assessment Long-term follow up of patients who received SAR421869 in a previous study TDU13600
- Primary Outcome Measures
Name Time Method The incidence of adverse events 15 years The number and percentage of patients with treatment emergent adverse events
- Secondary Outcome Measures
Name Time Method 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)
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
Trial Locations
- Locations (2)
Oregon Health and Science University Site Number : 840001
🇺🇸Portland, Oregon, United States
Investigational Site Number : 250001
🇫🇷Paris, France