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

• ClinicalTrials.gov Identifier: NCT02065011
• Recruitment Status: Active, not recruiting
• First Posted: February 17, 2014
• Last Update Posted: May 12, 2022
• Sponsor: Sanofi
• Information provided by (Responsible Party): Sanofi

Study Description

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

Condition or disease	Intervention/treatment	Phase
Usher's Syndrome	Drug: Blood draw for the laboratory assessment	Phase 1; Phase 2

Detailed Description:

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

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 9 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Other
• 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
• Actual Study Start Date: September 12, 2013
• Estimated Primary Completion Date: June 13, 2031
• Estimated Study Completion Date: June 13, 2031

Arms and Interventions

Arm	Intervention/treatment
Long-term follow up; Long-term follow up of patients who received SAR421869 in a previous study TDU13600	Drug: Blood draw for the laboratory assessment

Outcome Measures

Primary Outcome Measures :

1. The incidence of adverse events [ Time Frame: 15 years ]
   The number and percentage of patients with treatment emergent adverse events

Secondary Outcome Measures :

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)

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

United States, Oregon

Investigational Site Number 840001

Portland, Oregon, United States, 97239-3098

France

Investigational Site Number 250001

Paris, France, 75012

Sponsors and Collaborators

Sanofi

Investigators

• Study Director: Clinical Sciences & Operations, MD; Sanofi

More Information

• Responsible Party: Sanofi
• ClinicalTrials.gov Identifier: NCT02065011
• Other Study ID Numbers: LTS13619; 2013-000597-29 ( EudraCT Number )
• First Posted: February 17, 2014
• Last Update Posted: May 12, 2022
• Last Verified: May 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Usher Syndromes; Syndrome; Disease; Pathologic Processes; 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; Eye Diseases, Hereditary; Genetic Diseases, Inborn