BS01 in Patients With Retinitis Pigmentosa

NCT ID: NCT04278131

Last Updated: 2026-01-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-02-06

Study Completion Date

2029-12-30

Brief Summary

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A Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP (AAV2-CAG-ChronosFP) in Patients with Retinitis Pigmentosa

Detailed Description

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Conditions

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Retinitis Pigmentosa

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

BSO1 Cohort 1 dose

Group Type EXPERIMENTAL

BS01

Intervention Type DRUG

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

Cohort 2

BS01 Cohort 2 dose

Group Type EXPERIMENTAL

BS01

Intervention Type DRUG

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

Cohort 3

BS01 Cohort 3 dose

Group Type EXPERIMENTAL

BS01

Intervention Type DRUG

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

Cohort 4

BS01 Cohort4 dose

Group Type EXPERIMENTAL

BS01

Intervention Type DRUG

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

Cohort 5

BS01 Cohort4 dose

Group Type EXPERIMENTAL

BS01

Intervention Type DRUG

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

Cohort 6

Treat opposite eye

Group Type EXPERIMENTAL

BS01

Intervention Type DRUG

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

Interventions

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BS01

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

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Confirmed diagnosis of retinitis pigmentosa
* At least one eye with vision no better than 20/160 visual acuity on the EDTRS scale or, for patients with peripheral vision loss, a visual field no greater than 30°.

Exclusion Criteria

* Prior receipt of any AAV gene therapy product
* Large amplitude nystagmus
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Bionic Sight LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Sheila Nirenberg, PhD

Role: STUDY_DIRECTOR

Bionic Sight LLC

Locations

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New Jersey Retina

Teaneck, New Jersey, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Minako Koga

Role: CONTACT

Facility Contacts

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Lauryn Onkendi

Role: primary

(201)837-7300 ext. opt. 4

Other Identifiers

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BS01-RP-001

Identifier Type: -

Identifier Source: org_study_id

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