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

Phase 2Active Not RecruitingNCT02065011

Sanofi9 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

OTHER

Masking

NONE

Enrollment

Conditions

Usher's Syndrome

Interventions

Blood draw for the laboratory assessmentDRUG

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (2)

Oregon Health and Science University Site Number : 840001

Portland, Oregon, United States

Investigational Site Number : 250001

Paris, France

Outcomes

Primary Outcomes

The incidence of adverse events

The number and percentage of patients with treatment emergent adverse events

Time frame: 15 years

Secondary Outcomes

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

Time frame: baseline to 15 years

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)

Time frame: baseline to 15 years

Data from ClinicalTrials.gov

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