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

NCT02065011 | Active Not Recruiting Phase 2 FDA Drug

• 4 other identifiers: LTS136192024-513500-34US1/002/132013-000597-29
• Study Type: interventional
• Target: 9
• Locations: 2 countries
• Sites: 2

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

Conditions

Usher's Syndrome

Outcome Measures

Primary Outcomes (1)

• The incidence of adverse events

  The number and percentage of patients with treatment emergent adverse events

  15 years

Secondary Outcomes (2)

• Clinically important changes in ocular safety assessments

  baseline to 15 years

• Delay in retinal degeneration

  baseline to 15 years

Study Arms (1)

Long-term follow up

OTHER

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

Drug: Blood draw for the laboratory assessment

Interventions

Blood draw for the laboratory assessment DRUG

Long-term follow up

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• Did not receive SAR421869 as part of the TDU13600 protocol.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (2)

Oregon Health and Science University Site Number : 840001

Portland, Oregon, 97239-3098, United States

Location

Investigational Site Number : 250001

Paris, 75012, France

Location

Related Links

• LTS13619 Plain Language Results Summary

MeSH Terms

Conditions

Usher Syndromes

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Study Officials

• Clinical Sciences & Operations, MD

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 14, 2014
• First Posted: February 17, 2014
• Study Start: September 12, 2013
• Primary Completion (Estimated): June 13, 2031
• Study Completion (Estimated): June 13, 2031
• Last Updated: January 13, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share

Locations

US (1); FR (1)