Gene therapy for patients with retinitis pigmentosa
Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP in Patients With Retinitis Pigmentosa
This study is testing a new gene therapy to see if it can help people with retinitis pigmentosa who can only see light in one eye.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Bionic Sight LLC Industry-sponsored |
| Locations | 1 site (Teaneck, New Jersey) |
| Trial ID | NCT04278131 on ClinicalTrials.gov |
What this trial studies
This is a non-randomized, open-label Phase 1/2 dose escalation study evaluating BS01, a recombinant adeno-associated virus vector designed to express an enhanced light-sensitive channelrhodopsin gene. The study aims to assess the safety and efficacy of BS01 in patients diagnosed with retinitis pigmentosa who have bare light perception in at least one eye. Participants will receive varying doses of the treatment to determine the optimal dosage for therapeutic effect.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals with a confirmed diagnosis of retinitis pigmentosa and bare light perception in at least one eye.
Not a fit: Patients who have previously received any AAV gene therapy product or have large amplitude nystagmus may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could restore vision or improve light sensitivity in patients with retinitis pigmentosa.
How similar studies have performed: While gene therapy for retinal conditions is an emerging field, this specific approach using BS01 is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Confirmed diagnosis of retinitis pigmentosa * At least one eye with vision no better than 20/160 visual acuity on the EDTRS scale or, for patients with peripheral vision loss, a visual field no greater than 30°. Exclusion Criteria: * Prior receipt of any AAV gene therapy product * Large amplitude nystagmus
Where this trial is running
Teaneck, New Jersey
- New Jersey Retina — Teaneck, New Jersey, United States (Recruiting)
Study contacts
- Study coordinator: Minako Koga
- Email: mkoga@kmphc.com
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.