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

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

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

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Other
    • Masking: None (Open Label)
  • Study Primary Completion Date: June 2032

Detailed Description

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

Interventions

  • Drug: Blood draw for the laboratory assessment

Arms, Groups and Cohorts

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

Clinical Trial Outcome Measures

Primary Measures

  • The incidence of adverse events
    • Time Frame: 15 years
    • The number and percentage of patients with treatment emergent adverse events

Secondary Measures

  • 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
  • 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)

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Sanofi
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Clinical Sciences & Operations, MD, Study Director, Sanofi

Clinical trials entries are delivered from the US National Institutes of Health and are not reviewed separately by this site. Please see the identifier information above for retrieving further details from the government database.

At TrialBulletin.com, we keep tabs on over 200,000 clinical trials in the US and abroad, using medical data supplied directly by the US National Institutes of Health. Please see the About and Contact page for details.