Back to resultsBS01 in Patients With Retinitis Pigmentosa
Primary Purpose
Retinitis Pigmentosa
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
BS01
Sponsored by
About this trial
This is an interventional treatment trial for Retinitis Pigmentosa
Eligibility Criteria
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
Sites / Locations
- OCLIRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
Experimental
Experimental
Experimental
Arm Label
Cohort 1
Cohort 2
Cohort 3
Cohort 4
Arm Description
BSO1 Cohort 1 dose
BS01 Cohort 2 dose
BS01 Cohort 3 dose
BS01 Cohort4 dose
Outcomes
Primary Outcome Measures
Primary Outcome Measure
Number of subjects with adverse events, changes in hematology/chemistry
Secondary Outcome Measures
Secondary Outcome Measures
Changes in light detection by Diagnosys visual function testing
Changes in shape and motion detection by Diagnosys visual function testing
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04278131
Brief Title
BS01 in Patients With Retinitis Pigmentosa
Official Title
Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP in Patients With Retinitis Pigmentosa
Study Type
Interventional
2. Study Status
Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
February 6, 2020 (Actual)
Primary Completion Date
December 30, 2024 (Anticipated)
Study Completion Date
December 30, 2029 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bionic Sight LLC
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Retinitis Pigmentosa
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Cohort 1
Arm Type
Experimental
Arm Description
BSO1 Cohort 1 dose
Arm Title
Cohort 2
Arm Type
Experimental
Arm Description
BS01 Cohort 2 dose
Arm Title
Cohort 3
Arm Type
Experimental
Arm Description
BS01 Cohort 3 dose
Arm Title
Cohort 4
Arm Type
Experimental
Arm Description
BS01 Cohort4 dose
Intervention Type
Drug
Intervention Name(s)
BS01
Intervention Description
Non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector expressing an enhanced light-sensitive channelrhodopsin gene (ChronosFP)
Primary Outcome Measure Information:
Title
Primary Outcome Measure
Description
Number of subjects with adverse events, changes in hematology/chemistry
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Secondary Outcome Measures
Description
Changes in light detection by Diagnosys visual function testing
Changes in shape and motion detection by Diagnosys visual function testing
Time Frame
12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sheila Nirenberg, PhD
Organizational Affiliation
Bionic Sight LLC
Official's Role
Study Director
Facility Information:
Facility Name
OCLI
City
New York
State/Province
New York
ZIP/Postal Code
11572
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Clinical Trial Coordinator
Phone
516-593-4026
Email
ilezcano@ocli.net
First Name & Middle Initial & Last Name & Degree
Glenn Stoller, MD
12. IPD Sharing Statement
Plan to Share IPD
No
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BS01 in Patients With Retinitis Pigmentosa
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