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BS01 in Patients With Retinitis Pigmentosa

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04278131
Recruitment Status : Recruiting
First Posted : February 20, 2020
Last Update Posted : February 20, 2020
Sponsor:
Information provided by (Responsible Party):
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).

Condition or disease Intervention/treatment Phase
Retinitis Pigmentosa Drug: BS01 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP in Patients With Retinitis Pigmentosa
Actual Study Start Date : February 6, 2020
Estimated Primary Completion Date : June 30, 2022
Estimated Study Completion Date : June 30, 2026

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Cohort 1
BSO1 Cohort 1 dose
Drug: BS01
Non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector expressing an enhanced light-sensitive channelrhodopsin gene (ChronosFP)

Experimental: Cohort 2
BS01 Cohort 2 dose
Drug: BS01
Non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector expressing an enhanced light-sensitive channelrhodopsin gene (ChronosFP)

Experimental: Cohort 3
BS01 Cohort 3 dose
Drug: BS01
Non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector expressing an enhanced light-sensitive channelrhodopsin gene (ChronosFP)




Primary Outcome Measures :
  1. Primary Outcome Measure [ Time Frame: 12 months ]
    Number of subjects with adverse events, changes in hematology/chemistry


Secondary Outcome Measures :
  1. Secondary Outcome Measures [ Time Frame: 12 months ]
    1. Changes in light detection by Diagnosys visual function testing
    2. Changes in shape and motion detection by Diagnosys visual function testing



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04278131


Locations
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United States, New York
OCLI Recruiting
New York, New York, United States, 11572
Contact: Clinical Trial Coordinator    516-593-4026    ilezcano@ocli.net   
Principal Investigator: Glenn Stoller, MD         
Sponsors and Collaborators
Bionic Sight LLC
Investigators
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Study Director: Sheila Nirenberg, PhD Bionic Sight LLC
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Responsible Party: Bionic Sight LLC
ClinicalTrials.gov Identifier: NCT04278131    
Other Study ID Numbers: BS01-RP-001
First Posted: February 20, 2020    Key Record Dates
Last Update Posted: February 20, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn