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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

Tracking Information
First Submitted Date  ICMJE February 13, 2020
First Posted Date  ICMJE February 20, 2020
Last Update Posted Date February 20, 2020
Actual Study Start Date  ICMJE February 6, 2020
Estimated Primary Completion Date June 30, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 18, 2020)
Primary Outcome Measure [ Time Frame: 12 months ]
Number of subjects with adverse events, changes in hematology/chemistry
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: February 18, 2020)
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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE BS01 in Patients With Retinitis Pigmentosa
Official Title  ICMJE Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP in Patients With Retinitis Pigmentosa
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Retinitis Pigmentosa
Intervention  ICMJE Drug: BS01
Non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector expressing an enhanced light-sensitive channelrhodopsin gene (ChronosFP)
Study Arms  ICMJE
  • Experimental: Cohort 1
    BSO1 Cohort 1 dose
    Intervention: Drug: BS01
  • Experimental: Cohort 2
    BS01 Cohort 2 dose
    Intervention: Drug: BS01
  • Experimental: Cohort 3
    BS01 Cohort 3 dose
    Intervention: Drug: BS01
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 18, 2020)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 30, 2026
Estimated Primary Completion Date June 30, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04278131
Other Study ID Numbers  ICMJE BS01-RP-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Bionic Sight LLC
Study Sponsor  ICMJE Bionic Sight LLC
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Sheila Nirenberg, PhD Bionic Sight LLC
PRS Account Bionic Sight LLC
Verification Date February 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP