Retinitis PigmentosaPhase 1/2NCT04278131
BS01 in Patients With Retinitis Pigmentosa
A Phase 1/2 study testing BS01 in people with Retinitis Pigmentosa, run by Bionic Sight LLC at 1 site in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- BS01
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Feb 2020to 30 Dec 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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°.
Who cannot join
- Prior receipt of any AAV gene therapy product
- Large amplitude nystagmus
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Minako Koga contact
United States1 site, 1 recruiting
- Recruiting
New Jersey Retina
Teaneck, New Jersey, 07666
Lauryn Onkendi, (201)837-7300 ext. opt. 4, lonkendi@njretina.com
Paul Hahn, MD
About this study
Official title: Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP in Patients With Retinitis Pigmentosa
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
- Conditions
- Retinitis Pigmentosa
- Being tested
- BS01
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BS01Drug
Non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector expressing an enhanced light-sensitive channelrhodopsin gene (ChronosFP)
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | BSO1 Cohort 1 dose Receives: Drug: BS01 |
| Cohort 2 | Experimental | BS01 Cohort 2 dose Receives: Drug: BS01 |
| Cohort 3 | Experimental | BS01 Cohort 3 dose Receives: Drug: BS01 |
| Cohort 4 | Experimental | BS01 Cohort4 dose Receives: Drug: BS01 |
| Cohort 5 | Experimental | BS01 Cohort4 dose Receives: Drug: BS01 |
| Cohort 6 | Experimental | Treat opposite eye Receives: Drug: BS01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Outcome Measure
Number of subjects with adverse events, changes in hematology/chemistry
Measured: 12 months
Secondary outcomes (1)
Secondary Outcome Measures
Changes in light detection (threshold to detect) Changes in motion detection Changes in shape/object/letter detection Changes in performance on maze navigation task
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2020
- Study start6 Feb 2020
- Primary completion30 Dec 2026 (estimated)
- Study completion30 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bionic Sight LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sheila Nirenberg, PhD, Study director, Bionic Sight LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04278131
- Sponsor's protocol ID
- BS01-RP-001
- Status last verified
- Jan 2026
- First submitted
- 13 Feb 2020
- Last updated
- 23 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP in Patients With Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT04278131
- Acronym / sponsor ID
- BS01-RP-001
- Conditions
- Retinitis Pigmentosa
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Bionic Sight LLCIndustry (pharma, biotech, devices)
- Study start
- 6 Feb 2020 (actual)
- Primary completion
- 30 Dec 2026 (estimated)
- Study completion
- 30 Dec 2029 (estimated)
- First posted
- 20 Feb 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: BS01BSO1 Cohort 1 dose |
| Cohort 2 | Experimental | Drug: BS01BS01 Cohort 2 dose |
| Cohort 3 | Experimental | Drug: BS01BS01 Cohort 3 dose |
| Cohort 4 | Experimental | Drug: BS01BS01 Cohort4 dose |
| Cohort 5 | Experimental | Drug: BS01BS01 Cohort4 dose |
| Cohort 6 | Experimental | Drug: BS01Treat opposite eye |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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°.
Key exclusion criteria (2 listed)
- Prior receipt of any AAV gene therapy product
- Large amplitude nystagmus
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BS01Non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector expressing an enhanced light-sensitive channelrhodopsin gene (ChronosFP) | Drug | Cohort 1, Cohort 2, Cohort 3, Cohort 4, Cohort 5, Cohort 6 |
Primary outcome
Primary Outcome Measure
Time frame: 12 months
Number of subjects with adverse events, changes in hematology/chemistry
1 secondary outcome
Secondary Outcome Measures
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Minako Koga contact
Sheila Nirenberg, PhD study director
Bionic Sight LLC
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing BS01 in people with Retinitis Pigmentosa, run by Bionic Sight LLC at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 23 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Minako Koga (mkoga@kmphc.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Outcome Measure (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jan 2026.