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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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
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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 |
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 |

Arm | Intervention/treatment |
---|---|
Long-term follow up
Long-term follow up of patients who received SAR421869 in a previous study TDU13600
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Drug: Blood draw for the laboratory assessment |
- The incidence of adverse events [ Time Frame: 15 years ]The number and percentage of patients with treatment emergent adverse events
- 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)

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.
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.

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
United States, Oregon | |
Investigational Site Number 840001 | |
Portland, Oregon, United States, 97239-3098 | |
France | |
Investigational Site Number 250001 | |
Paris, France, 75012 |
Study Director: | Clinical Sciences & Operations, MD | Sanofi |
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 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
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 |