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

Sanofi

Status and phase: Active, not recruiting; Phase 2

Conditions: Usher's Syndrome

Treatments: Drug: Blood draw for the laboratory assessment

Study type: Interventional

Funder types: Industry

Identifiers

NCT02065011

LTS13619

2013-000597-29 (EudraCT Number)

Details and patient eligibility

About

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

Full description

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

Enrollment: 9 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Other

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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9 participants in 1 patient group

Long-term follow up

Other group

Description:

Long-term follow up of patients who received SAR421869 in a previous study TDU13600

Treatment:

Drug: Blood draw for the laboratory assessment