SAR421869 for Usher Syndrome

Not currently recruiting at 3 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Sanofi

Disqualifiers: Not in TDU13600, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to assess the long-term safety and effectiveness of SAR421869 for individuals with Usher syndrome Type 1B, a condition affecting hearing and vision. The researchers seek to evaluate treatment tolerance over time and understand its ongoing impact. This trial specifically targets participants from an earlier study who received SAR421869 via a special eye injection. Those who participated in that prior study might be suitable candidates. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications.

Is there any evidence suggesting that SAR421869 is likely to be safe for humans?

Research has shown that SAR421869 has been tested for safety in people with Usher syndrome Type 1B. In these studies, researchers administered the treatment through subretinal injections, meaning injections under the retina, the light-sensitive part at the back of the eye. The main goal was to assess how well people could tolerate the treatment over time.

Previous patients have generally tolerated SAR421869 well. Long-term studies have focused on monitoring any side effects. While detailed results are not fully available yet, ongoing follow-ups suggest no major safety concerns. This indicates that SAR421869 is relatively safe for people with this condition.

Since this study is in a later phase, earlier research has already provided some confidence in its safety. However, like any clinical trial, some risk remains, and it is important to consider this information and discuss it with a healthcare provider. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Usher Syndrome, which primarily focus on managing symptoms through hearing aids and cochlear implants, SAR421869 represents a potential breakthrough by directly targeting the underlying genetic cause of the condition. Researchers are excited because SAR421869 is designed to deliver a gene therapy specifically to the retina, which might help restore or preserve vision in patients. This innovative approach could offer a more comprehensive solution, addressing not just the symptoms but the root cause of vision loss in Usher Syndrome.

What evidence suggests that SAR421869 might be an effective treatment for Usher syndrome?

Research has shown that SAR421869 has been tested for Usher syndrome Type 1B, primarily to assess its safety and tolerability. In early studies, patients received the treatment through eye injections. Results showed no major improvements in vision for up to four years post-treatment. Some participants experienced a gradual decline in vision after four years. This indicates that while SAR421869 remains under study, there is currently limited evidence of its effectiveness in improving vision for Usher syndrome Type 1B. However, ongoing research, including this trial's long-term follow-up, aims to better understand its potential benefits and long-term effects. ^{2 6 7 8 9}

Who Is on the Research Team?

Clinical Sciences & Operations, MD

Principal Investigator

Sanofi

Are You a Good Fit for This Trial?

This trial is for patients with Usher Syndrome Type 1B who previously enrolled in the TDU13600 protocol and received a subretinal injection of SAR421869. Participants must have given written consent. Those who did not receive SAR421869 as part of the earlier TDU13600 study cannot join.

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).

I have received a SAR421869 injection in my eye.

Must have been enrolled in protocol TDU13600.

Exclusion Criteria

You have not previously received SAR421869 as part of the TDU13600 protocol.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Long-term follow-up

Long-term follow-up of patients who received SAR421869 in a previous study TDU13600

15 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• SAR421869

Trial Overview The study focuses on long-term safety, tolerability, and biological activity of SAR421869 in individuals with Usher Syndrome Type 1B. It involves regular blood draws to monitor these aspects over an extended period.

How Is the Trial Designed?

Group I: Long-term follow up Experimental Treatment 1 Intervention

Long-term follow up of patients who received SAR421869 in a previous study TDU13600

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

Published Research Related to This Trial

Lifitegrast ophthalmic solution 5.0% was found to be safe and well tolerated in a study involving 2464 participants, with most treatment-emergent adverse events being mild to moderate in severity.

The treatment significantly improved drop comfort within 3 minutes of instillation, indicating a quick onset of relief for patients with dry eye disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and tolerability of lifitegrast ophthalmic solution 5.0%: Pooled analysis of five randomized controlled trials in dry eye disease. Nichols, KK., Donnenfeld, ED., Karpecki, PM., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT01505062

NCT01505062 | Study of SAR421869 in Participants With ... To evaluate the safety and tolerability of ascending doses of subretinal injections of SAR421869 in participants with Usher syndrome type 1B. To evaluate for ...

2. iovs.arvojournals.org iovs.arvojournals.org/article.aspx?articleid=2794295

Six-year results of a Phase I/IIa gene therapy trial in patients ... Thus, none of the subjects had any significant improvements from baseline up to year 4. Some subjects had a slow significant decline after year 4, likely due to ...

3. withpower.com withpower.com/trial/phase-2-usher-syndromes-8-2013-25d27

SAR421869 for Usher Syndrome · Info for Participants This trial is for patients with Usher Syndrome Type 1B who previously enrolled in the TDU13600 protocol and received a subretinal injection of SAR421869.

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6789203/

bone marrow derived stem cells in the treatment of Usher ... Of the eyes that improved the average increase in visual acuity was 36.4% on LogMAR with improvements ranging from 23% to 94%. The average post- ...

5. malacards.org malacards.org/card/usher_syndrome

Usher Syndrome (USH) Usher syndrome is a rare genetic disorder that causes sensorineural hearing loss and retinitis pigmentosa, leading to progressive vision loss.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT02065011

NCT02065011 | A Study to Determine the Long-Term ... A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B. ClinicalTrials.gov ID ...

7. clinicaltrials.eu clinicaltrials.eu/trial/study-on-long-term-safety-and-effects-of-sar421869-for-patients-with-usher-syndrome-type-1b/

Study on Long-Term Safety and Effects of SAR421869 for ... This clinical trial evaluates the long-term safety and effects of SAR421869, a gene therapy, on patients diagnosed with Usher Syndrome Type 1B, ...

8. app.emergingmed.com app.emergingmed.com/emed/trial/60978

Study of SAR421869 in Participants With Retinitis ... At the end of the study, the participants were invited to enter in an open-label safety study for long-term follow-up visits (at least once every six months) ...

9. ctv.veeva.com ctv.veeva.com/study/a-study-to-determine-the-long-term-safety-tolerability-and-biological-activity-of-sar421869-in-pati

A Study to Determine the Long-Term Safety, Tolerability and ... Primary Objective: To evaluate the long-term safety and tolerability of SAR421869 in patients with Usher syndrome Type 1B.