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

May 9, 2022 updated by: Sanofi

An Open-label 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

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

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

Study Type

Interventional

Enrollment (Anticipated)

9

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Paris, France, 75012
        • Investigational Site Number 250001
  • United States
    • Oregon
      • Portland, Oregon, United States, 97239-3098
        • Investigational Site Number 840001

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: 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

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
baseline to 15 years
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)
baseline to 15 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sanofi

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 12, 2013

Primary Completion (Anticipated)

June 13, 2031

Study Completion (Anticipated)

June 13, 2031

Study Registration Dates

First Submitted

February 14, 2014

First Submitted That Met QC Criteria

February 14, 2014

First Posted (Estimate)

February 17, 2014

Study Record Updates

Last Update Posted (Actual)

May 12, 2022

Last Update Submitted That Met QC Criteria

May 9, 2022

Last Verified

May 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • LTS13619
  • 2013-000597-29 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD 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

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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