BS01 in Patients With Retinitis Pigmentosa

Phase 1

Recruiting

• Conditions: Retinitis Pigmentosa
• Interventions: Drug: BS01

• Registration Number: NCT04278131
• Lead Sponsor: Bionic Sight LLC

Brief Summary

Non-randomized, open label, Phase 1/2 dose escalation study of BS01, a non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector expressing an enhanced light-sensitive channelrhodopsin gene (ChronosFP).

Detailed Description

Not available

Study Timeline

• Study Start: 2020-02-06
• Study First Submitted: 2020-02-13
• Study First Submitted QC: 2020-02-18
• Study First Posted: 2020-02-20
• Status Verified: 2023-05
• Last Update Submitted: 2023-05-01
• Last Update Posted: 2023-05-03
• Primary Completion: 2024-12-30
• Study Completion: 2029-12-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

• Confirmed diagnosis of retinitis pigmentosa
• Bare light perception in at least one eye

Exclusion Criteria

• Prior receipt of any AAV gene therapy product
• Large amplitude nystagmus

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 1	BS01	BSO1 Cohort 1 dose
Cohort 2	BS01	BS01 Cohort 2 dose
Cohort 3	BS01	BS01 Cohort 3 dose
Cohort 4	BS01	BS01 Cohort4 dose

Primary Outcome Measures

Name	Time	Method
Primary Outcome Measure	12 months	Number of subjects with adverse events, changes in hematology/chemistry

Secondary Outcome Measures

Name	Time	Method
Secondary Outcome Measures	12 months	1. Changes in light detection by Diagnosys visual function testing; 2. Changes in shape and motion detection by Diagnosys visual function testing

Trial Locations

Locations (1)

OCLI; 🇺🇸 New York, New York, United States