SAR421869 for Usher Syndrome

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

9 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

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

What safety data exists for SAR421869 (sepofarsen) in humans?

In a trial for a different eye condition, sepofarsen was generally safe but caused some eye-related side effects. Most side effects were mild, but some patients developed cataracts that required lens replacement. ^{1 2 3 4 5}

How is the drug SAR421869 unique for treating Usher Syndrome?

SAR421869 is a novel treatment for Usher Syndrome, a condition with no currently approved therapies for its retinal degeneration aspect. Unlike existing options that focus on managing hearing loss with devices like hearing aids and cochlear implants, SAR421869 may target the underlying genetic causes of the syndrome, potentially offering a new approach to treatment. ^{6 7 8 9 10}

What is the purpose of this trial?

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

Research Team

Clinical Sciences & Operations, MD

Principal Investigator

Sanofi

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

References

1. United States 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. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial. [2022]

3. Netherlands pubmed.ncbi.nlm.nih.gov

TNF-α inhibitor tanfanercept (HBM9036) improves signs and symptoms of dry eye in a phase 2 trial in the controlled adverse environment in China. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Topical administration of interleukin-1 receptor antagonist as a therapy for aqueous-deficient dry eye in autoimmune disease. [2021]

5. United States pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Carbomer-Based Lipid-Containing Artificial Tear Formulations in Patients With Dry Eye Syndrome. [2022]

6. China pubmed.ncbi.nlm.nih.gov

[Genetic analysis and prenatal diagnosis of a Chinese pedigree affected with Usher syndrome due to novel compound heterozygous variants of PCDH15 gene]. [2022]

7. Denmark pubmed.ncbi.nlm.nih.gov

Evaluation of visual impairment in Usher syndrome 1b and Usher syndrome 2a. [2019]

8. United States pubmed.ncbi.nlm.nih.gov

Retinal disease in Usher syndrome III caused by mutations in the clarin-1 gene. [2022]

9. United States pubmed.ncbi.nlm.nih.gov

Usher syndrome: clinical features, molecular genetics and advancing therapeutics. [2021]

10. England pubmed.ncbi.nlm.nih.gov

Development of a genotyping microarray for Usher syndrome. [2022]