NCT04278131

Brief Summary

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

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at P25-P50 for phase_1

Timeline
45mo left

Started Feb 2020

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress63%
Feb 2020Dec 2029

Study Start

First participant enrolled

February 6, 2020

Completed
7 days until next milestone

First Submitted

Initial submission to the registry

February 13, 2020

Completed
7 days until next milestone

First Posted

Study publicly available on registry

February 20, 2020

Completed
6.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 30, 2026

Expected
3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 30, 2029

Last Updated

January 23, 2026

Status Verified

January 1, 2026

Enrollment Period

6.9 years

First QC Date

February 13, 2020

Last Update Submit

January 21, 2026

Conditions

Retinitis Pigmentosa

Outcome Measures

Primary Outcomes (1)

  • Primary Outcome Measure

    Number of subjects with adverse events, changes in hematology/chemistry

    12 months

Secondary Outcomes (1)

  • Secondary Outcome Measures

    12 months

Study Arms (6)

Cohort 1

EXPERIMENTAL

BSO1 Cohort 1 dose

Drug: BS01

Cohort 2

EXPERIMENTAL

BS01 Cohort 2 dose

Drug: BS01

Cohort 3

EXPERIMENTAL

BS01 Cohort 3 dose

Drug: BS01

Cohort 4

EXPERIMENTAL

BS01 Cohort4 dose

Drug: BS01

Cohort 5

EXPERIMENTAL

BS01 Cohort4 dose

Drug: BS01

Cohort 6

EXPERIMENTAL

Treat opposite eye

Drug: BS01

Interventions

BS01DRUG

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

Cohort 1Cohort 2Cohort 3Cohort 4Cohort 5Cohort 6

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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°.

You may not qualify if:

  • Prior receipt of any AAV gene therapy product
  • Large amplitude nystagmus

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

New Jersey Retina

Teaneck, New Jersey, 07666, United States

RECRUITING

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal DystrophiesRetinal DegenerationRetinal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Sheila Nirenberg, PhD

    Bionic Sight LLC

    STUDY DIRECTOR

Central Study Contacts

Minako Koga

CONTACT

mkoga@kmphc.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 13, 2020

First Posted

February 20, 2020

Study Start

February 6, 2020

Primary Completion (Estimated)

December 30, 2026

Study Completion (Estimated)

December 30, 2029

Last Updated

January 23, 2026

Record last verified: 2026-01

Data Sharing

IPD Sharing
Will not share

Locations

US(1)