A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B

Usher's Syndrome Clinical Trial

Official title:

An Open-label Study to Determine the Long-term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02065011
• Other study ID #: LTS13619
• Secondary ID: 2013-000597-29
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: September 12, 2013
• Est. completion date: June 13, 2031

Study information

• Verified date: November 2023
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Description:

The total duration of study period is up to 15 years.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 9
• Est. completion date: June 13, 2031
• Est. primary completion date: June 13, 2031
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Syndrome
• Usher Syndromes
• Usher's Syndrome

Intervention

Drug:
• Blood draw for the laboratory assessment

Locations

Country	Name	City	State
France	Investigational Site Number : 250001	Paris
United States	Oregon Health and Science University Site Number : 840001	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Sanofi

Countries where clinical trial is conducted

United States,  France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The incidence of adverse events	The number and percentage of patients with treatment emergent adverse events	15 years
Secondary	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
Secondary	Delay in retinal degeneration	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