Back to resultsA Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B
Primary Purpose
Usher's Syndrome
Status
Active
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
Blood draw for the laboratory assessment
Sponsored by
About this trial
This is an interventional other trial for Usher's Syndrome
Eligibility 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.
Sites / Locations
- Investigational Site Number 840001
- Investigational Site Number 250001
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
Long-term follow up
Arm Description
Long-term follow up of patients who received SAR421869 in a previous study TDU13600
Outcomes
Primary Outcome Measures
The incidence of adverse events
The number and percentage of patients with treatment emergent adverse events
Secondary Outcome Measures
Clinically important changes in ocular safety assessments
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
Measured as change from baseline in function relative to untreated contralateral eye on: BCVA, static perimetry, autofluorescence, optical coherence tomography (OCT)
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02065011
Brief Title
A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B
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
Study Type
Interventional
2. Study Status
Record Verification Date
May 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 12, 2013 (Actual)
Primary Completion Date
June 13, 2031 (Anticipated)
Study Completion Date
June 13, 2031 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sanofi
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. 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
Detailed Description
The total duration of study period is up to 15 years.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Usher's Syndrome
7. Study Design
Primary Purpose
Other
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
9 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Long-term follow up
Arm Type
Other
Arm Description
Long-term follow up of patients who received SAR421869 in a previous study TDU13600
Intervention Type
Drug
Intervention Name(s)
Blood draw for the laboratory assessment
Primary Outcome Measure Information:
Title
The incidence of adverse events
Description
The number and percentage of patients with treatment emergent adverse events
Time Frame
15 years
Secondary Outcome Measure Information:
Title
Clinically important changes in ocular safety assessments
Description
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
Time Frame
baseline to 15 years
Title
Delay in retinal degeneration
Description
Measured as change from baseline in function relative to untreated contralateral eye on: BCVA, static perimetry, autofluorescence, optical coherence tomography (OCT)
Time Frame
baseline to 15 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Sciences & Operations, MD
Organizational Affiliation
Sanofi
Official's Role
Study Director
Facility Information:
Facility Name
Investigational Site Number 840001
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239-3098
Country
United States
Facility Name
Investigational Site Number 250001
City
Paris
ZIP/Postal Code
75012
Country
France
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing 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
Learn more about this trial
A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B
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