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NCT Number: NCT02065011

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

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Usher's Syndrome Abnormalities, Multiple Blindness Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Deaf-Blind Disorders Deafness Ear Diseases Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Hearing Disorders Hearing Loss Hearing Loss, Sensorineural Nervous System Diseases Neurologic Manifestations Otorhinolaryngologic Diseases Pathological Conditions, Signs and Symptoms Retinal Degeneration Retinal Diseases Retinal Dystrophies Retinitis Pigmentosa Sensation Disorders Signs and Symptoms Usher Syndromes Vision Disorders

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Investigational Site Number : 250001, Paris, France

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About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Blood draw for the laboratory assessment

Drug

Primary outcomes

  1. The incidence of adverse events

    Time frame: 15 years

    The number and percentage of patients with treatment emergent adverse events

Secondary outcomes

  1. Clinically important changes in ocular safety assessments

    Time frame: 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

  2. Delay in retinal degeneration

    Time frame: 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)

Sponsors and collaborators

Lead sponsor

Sanofi

Industry

Registry information

Official study title

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

Important dates

Study start
2013
Primary completion
2031
Study completion
2031
First posted
Feb 17, 2014
Registry last updated
Jan 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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