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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02065011
Recruitment Status : Active, not recruiting
First Posted : February 17, 2014
Last Update Posted : May 12, 2022
Information provided by (Responsible Party):

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.

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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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Usher Syndrome

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

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)

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02065011

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United States, Oregon
Investigational Site Number 840001
Portland, Oregon, United States, 97239-3098
Investigational Site Number 250001
Paris, France, 75012
Sponsors and Collaborators
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Study Director: Clinical Sciences & Operations, MD Sanofi
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Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT02065011    
Other Study ID Numbers: LTS13619
2013-000597-29 ( EudraCT Number )
First Posted: February 17, 2014    Key Record Dates
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Usher Syndromes
Pathologic Processes
Deaf-Blind Disorders
Hearing Loss
Hearing Disorders
Ear Diseases
Otorhinolaryngologic Diseases
Hearing Loss, Sensorineural
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases
Vision Disorders
Retinitis Pigmentosa
Retinal Dystrophies
Retinal Degeneration
Retinal Diseases
Eye Diseases
Abnormalities, Multiple
Congenital Abnormalities
Eye Diseases, Hereditary
Genetic Diseases, Inborn