Recruiting NCT04278131
BS01 in Patients With Retinitis Pigmentosa
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: BS01.
- Who it may be relevant to
- Registry conditions: Retinitis Pigmentosa. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP in Patients With Retinitis Pigmentosa
Overview
A Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP (AAV2-CAG-ChronosFP) in Patients with Retinitis Pigmentosa
Interventions
- Drug BS01
Non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector expressing an enhanced light-sensitive channelrhodopsin gene (ChronosFP)
Primary outcome measures
- Primary Outcome Measure [Time frame: 12 months]
Secondary outcome measures (1)
- Secondary Outcome Measures [Time frame: 12 months]
Eligibility 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 1 center
- New Jersey Retina — Teaneck
Identifiers
NCT: NCT04278131 · BS01-RP-001