BS01 in Patients With Retinitis Pigmentosa

Overview

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).

Full Title of Study: “Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP in Patients With Retinitis Pigmentosa”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Sequential Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: June 30, 2022

Interventions

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

Arms, Groups and Cohorts

  • Experimental: Cohort 1
    • BSO1 Cohort 1 dose
  • Experimental: Cohort 2
    • BS01 Cohort 2 dose
  • Experimental: Cohort 3
    • BS01 Cohort 3 dose

Clinical Trial Outcome Measures

Primary Measures

  • Primary Outcome Measure
    • Time Frame: 12 months
    • Number of subjects with adverse events, changes in hematology/chemistry

Secondary Measures

  • Secondary Outcome Measures
    • Time Frame: 12 months
    • Changes in light detection by Diagnosys visual function testing Changes in shape and motion detection by Diagnosys visual function testing

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Bionic Sight LLC
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Sheila Nirenberg, PhD, Study Director, Bionic Sight LLC

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