Usher's SyndromePhase 2NCT02065011
A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B
A Phase 2 study testing Blood draw for the laboratory assessment in people with Usher's Syndrome, run by Sanofi at 2 sites in 2 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Usher's Syndrome
- What is being tested?
- Blood draw for the laboratory assessment
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 12 Sep 2013to 13 Jun 2031
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Did not receive SAR421869 as part of the TDU13600 protocol.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
Oregon Health and Science University Site Number : 840001
Portland, Oregon, 97239-3098
About this study
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
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
Read the detailed description
The total duration of study period is up to 15 years.
- Conditions
- Usher's Syndrome
- Being tested
- Blood draw for the laboratory assessment
- Medical subject headings
- Usher Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood draw for the laboratory assessmentDrug
| Study group | Role | What they receive |
|---|---|---|
| Long-term follow up | Other | Long-term follow up of patients who received SAR421869 in a previous study TDU13600 Receives: Drug: Blood draw for the laboratory assessment |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence of adverse events
The number and percentage of patients with treatment emergent adverse events
Measured: 15 years
Secondary outcomes (2)
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
Measured: baseline to 15 years
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)
Measured: baseline to 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2014 (estimated)
- Study start12 Sep 2013
- Primary completion13 Jun 2031 (estimated)
- Study completion13 Jun 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Usher's SyndromeStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, MD, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02065011
- Sponsor's protocol ID
- LTS13619
- Other IDs
- 2024-513500-34, US1/002/13, 2013-000597-29
- Status last verified
- Jan 2026
- First submitted
- 14 Feb 2014
- Last updated
- 13 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02065011
- Acronym / sponsor ID
- LTS13619
- Conditions
- Usher's Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 9 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 12 Sep 2013 (actual)
- Primary completion
- 13 Jun 2031 (estimated)
- Study completion
- 13 Jun 2031 (estimated)
- First posted
- 17 Feb 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Long-term follow up | Other | Drug: Blood draw for the laboratory assessmentLong-term follow up of patients who received SAR421869 in a previous study TDU13600 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (1 listed)
- Did not receive SAR421869 as part of the TDU13600 protocol.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Blood draw for the laboratory assessment | Drug | Long-term follow up |
Primary outcome
The incidence of adverse events
Time frame: 15 years
The number and percentage of patients with treatment emergent adverse events
2 secondary outcomes
Clinically important changes in ocular safety assessments
Time frame: baseline to 15 years
Delay in retinal degeneration
Time frame: baseline to 15 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jan 2026.
- LTS13619 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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
Related trials for Usher's Syndrome
RecruitingPhase 1/2
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RecruitingPhase 3
A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
Retinitis Pigmentosa (RP), Usher Syndrome
Not yet recruitingPhase 2
Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
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Active, not recruitingObservational study
Rate of Progression in USH2A-related Retinal Degeneration
Usher Syndrome, Type 2A, Retinitis Pigmentosa 39
Active, not recruitingPhase 2
Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
Retinitis Pigmentosa (RP), Usher Syndrome Type 2
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations, MD study director
Sanofi
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Blood draw for the laboratory assessment in people with Usher's Syndrome, run by Sanofi at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 13 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence of adverse events (time frame: 15 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jan 2026.