Retinitis Pigmentosa (RP)Phase 1/2NCT02320812

Safety of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Retinitis Pigmentosa

A Phase 1/2 study testing human retinal progenitor cells in people with Retinitis Pigmentosa (RP), run by jCyte, Inc at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This study evaluates the safety and potential activity of a single dose of live human retinal progenitor cells (jCell) administered to adults with retinitis pigmentosa. Four different dose levels of cells will be assessed in each of two groups of patients. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Retinitis Pigmentosa (RP)
What is being tested?: human retinal progenitor cells
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 28 participants
Where: 2 sitesUnited States
Study dates: Jun 2015to 19 Jul 2017
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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

Clinical diagnosis of RP confirmed by electroretinogram (ERG) and willing to consent to mutation typing, if not already done
Best corrected visual acuity (BCVA) 20/63 or worse and no worse than hand motions (HM)
Adequate organ function and negative infectious disease screen
Female of childbearing potential must have negative pregnancy test and be willing to use medically accepted methods of contraception throughout the study

Who cannot join

Eye disease other than RP that impairs visual function
Pseudo-RP, cancer-associated retinopathies
History of malignancy or other end-stage organ disease, or any chronic disease requiring continuous treatment with system steroids, anticoagulants or immunosuppressive agents
Known allergy to penicillin or streptomycin

Trial locations and contacts

2 sites in 1 country.

United States2 sites

The Gavin Herbert Eye Institute, Univ California Irvine
Irvine, California
Retina-Vitreous Associates Medical Group
Los Angeles, California, 90074

Other trials in United States

About this study

Official title: A Prospective, Multicenter, Open-Label, Single-Arm Study of the Safety and Tolerability of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)

Read the detailed description

The primary purpose of this study is to test the safety and tolerability of the administration of a single dose of jCell to adults with retinitis pigmentosa.

The goal of jCell therapy is to preserve vision by intervening in the disease at a time when host photoreceptors can be protected and potentially reactivated.

Human allogeneic retinal progenitor cells will be injected into adults with advanced RP to see if the procedure is safe, if the cells survive, and whether they have any impact on the visual status of the patients.

Conditions: Retinitis Pigmentosa (RP)
Being tested: human retinal progenitor cells
Medical subject headings: Retinitis Pigmentosa

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

human retinal progenitor cellsBiological
Also called jCell
single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)

Study groups in NCT02320812
Study group	Role	What they receive
Treated subjects	Experimental	human retinal progenitor cells Receives: Biological: human retinal progenitor cells

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Subjects With Adverse Events as a Measure of Safety and Tolerability
proportion of subjects with treatment emergent adverse events (TEAE), related TEAE and severe TEAE
Measured: 12 months

Secondary outcomes (1)

Change in Mean Best Corrected Visual Acuity (BCVA)
change in mean best corrected visual acuity in test eye versus untreated eye; BCVA is measured using an eye chart and is reported as the number of letters read correctly (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). A positive change from baseline indicates an improvement and a negative change from baseline indicates a worsening. Change between baseline and month 12 is reported.
Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jun 201519 Jul 2017

First posted19 Dec 2014 (estimated)
Study startJun 2015
Primary completion19 Jul 2017
Study completion19 Jul 2017

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Retinitis Pigmentosa (RP)Studies 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: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 28 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02320812
Sponsor's protocol ID: JC-01
Status last verified: Mar 2019
First submitted: 16 Dec 2014
Last updated: 5 Mar 2019
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Individual data sharing: No

Publications

Yang J, Kuppermann BD, Liao D, Mehta MC, Hsiang C, Menges S, Boyer DS, Klassen H. Retinal progenitor cells (jCell) for retinitis pigmentosa. Front Cell Neurosci. 2025 Aug 25;19:1646156. doi: 10.3389/fncel.2025.1646156. eCollection 2025.
PubMed 40927136

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Recruiting
Brain Stimulation Effects on Orientation and Mobility Skills in Adults With Vision Impairment
Retinitis Pigmentosa (RP), Rod Cone Dystrophy
RecruitingPhase 2/3
Study to Evaluate the Efficacy of a Single Intravitreal Injection of RTx-015 in Participants With Retinitis Pigmentosa (PERCEPT)
Retinitis Pigmentosa (RP), Inherited Retinal Diseases
Active, not recruitingPhase 1/2
Intravitreal Adalimumab in Inherited and Degenerative Retinal Diseases
Retinitis Pigmentosa (RP), Extensive Macular Atrophy With Pseudodrusen (EMAP)
RecruitingPhase 1/2
A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
Primary Photoreceptor Disease, Retinitis Pigmentosa (RP)
RecruitingPhase 3
A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
Retinitis Pigmentosa (RP), Usher Syndrome

All Retinitis Pigmentosa (RP) trials

Study at a glance

From the registry record, last updated by the sponsor on 5 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Prospective, Multicenter, Open-Label, Single-Arm Study of the Safety and Tolerability of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)
ClinicalTrials.gov ID: NCT02320812
Acronym / sponsor ID: JC-01
Conditions: Retinitis Pigmentosa (RP)
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 28 participants (actual)
Lead sponsor: jCyte, IncIndustry (pharma, biotech, devices)
Collaborators: California Institute for Regenerative Medicine (CIRM)
Study start: Jun 2015
Primary completion: 19 Jul 2017 (actual)
Study completion: 19 Jul 2017 (actual)
First posted: 19 Dec 2014 (estimated)
Results: Posted 5 Mar 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

The primary purpose of this study is to test the safety and tolerability of the administration of a single dose of jCell to adults with retinitis pigmentosa.

The goal of jCell therapy is to preserve vision by intervening in the disease at a time when host photoreceptors can be protected and potentially reactivated.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Treated subjects	Experimental	Biological: human retinal progenitor cellshuman retinal progenitor cells

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Clinical diagnosis of RP confirmed by electroretinogram (ERG) and willing to consent to mutation typing, if not already done
Best corrected visual acuity (BCVA) 20/63 or worse and no worse than hand motions (HM)
Adequate organ function and negative infectious disease screen
Female of childbearing potential must have negative pregnancy test and be willing to use medically accepted methods of contraception throughout the study

Key exclusion criteria (4 listed)

Eye disease other than RP that impairs visual function
Pseudo-RP, cancer-associated retinopathies
History of malignancy or other end-stage organ disease, or any chronic disease requiring continuous treatment with system steroids, anticoagulants or immunosuppressive agents
Known allergy to penicillin or streptomycin

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
human retinal progenitor cellsAlso: jCellsingle intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)	Biological	Treated subjects

Primary outcome

Number of Subjects With Adverse Events as a Measure of Safety and Tolerability
Time frame: 12 months
proportion of subjects with treatment emergent adverse events (TEAE), related TEAE and severe TEAE

1 secondary outcome

Change in Mean Best Corrected Visual Acuity (BCVA)
Time frame: 12 months

Results and safety

Results posted to the registry on 5 Mar 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Treated Subjects	28	28

Primary outcome: Number of Subjects With Adverse Events as a Measure of Safety and Tolerability

Time frame: 12 months. Count of participants. Unit: Participants

Measure	Treated Subjects n = 28
Subjects with TEAE	25
Subjects with related TEAE	21
Subjects with grade 3 TEAE	1

Population analysed: all treated subjects

1 secondary outcome is reported in the full results.

Adverse events

Time frame: one year following single intravitreal injection of hRPC. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Treated Subjects	1 of 28 (3.6%)	20 of 28 (71.4%)	0 of 28

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2019.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 40927136Yang J, Kuppermann BD, Liao D, Mehta MC, Hsiang C, Menges S, Boyer DS, Klassen H. Retinal progenitor cells (jCell) for retinitis pigmentosa. Front Cell Neurosci. 2025 Aug 25;19:1646156. doi: 10.3389/fncel.2025.1646156. eCollection 2025.
Individual participant dataSharing planned: No

Related trials for Retinitis Pigmentosa (RP)

Recruiting
Brain Stimulation Effects on Orientation and Mobility Skills in Adults With Vision Impairment
Retinitis Pigmentosa (RP), Rod Cone Dystrophy
RecruitingPhase 2/3
Study to Evaluate the Efficacy of a Single Intravitreal Injection of RTx-015 in Participants With Retinitis Pigmentosa (PERCEPT)
Retinitis Pigmentosa (RP), Inherited Retinal Diseases
Active, not recruitingPhase 1/2
Intravitreal Adalimumab in Inherited and Degenerative Retinal Diseases
Retinitis Pigmentosa (RP), Extensive Macular Atrophy With Pseudodrusen (EMAP)
RecruitingPhase 1/2
A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
Primary Photoreceptor Disease, Retinitis Pigmentosa (RP)
RecruitingPhase 3
A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
Retinitis Pigmentosa (RP), Usher Syndrome

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. This study is not recruiting at the moment.

Baruch Kuppermann, MD principal investigator

Gavin Herbert Eye Institute, UCI, Irvine, CA

2 sites 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 human retinal progenitor cells in people with Retinitis Pigmentosa (RP), run by jCyte, Inc at 2 sites 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 4 inclusion and 4 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: finished, as of the sponsor's last update on 5 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites 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?

Yes. Results are posted in the registry (first posted 5 Mar 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Subjects With Adverse Events as a Measure of Safety and Tolerability (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02320812, which also lists 1 linked publication.

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 5 Mar 2019.