Retinitis PigmentosaPhase 2NCT06912633
Safety of a Single, Intravitreal Injection of 6.0M jCell (Famzeretcel) in Retinitis Pigmentosa (RP)
A Phase 2 study testing human retinal progenitor cells and Mock injection in people with Retinitis Pigmentosa, run by jCyte, Inc at 12 sites in United States.
Trial summary
This study evaluates the safety of a single injection of jCell (famzeretcel) comprising 6.0 million (6.0M) retinal progenitor cells over a six-month study period in a cohort of adult subjects with RP. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- human retinal progenitor cells and Mock injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 31 participants
- Where
- 12 sitesUnited States
- Study dates
- 20 Jun 2025to Dec 2026
- Phase
- Phase 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 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Clinical diagnosis of RP supported by at least 2 of the following clinical findings: (1) Loss of peripheral vision on formal visual field testing, (2) Symptoms of night blindness or difficulty adjusting to dim light, or (3) Optical coherence tomography (OCT) outer retinal atrophy consistent with RP.
- Electroretinography (ERG) results that support diagnosis of RP including nondetectable or severely reduced rod responses (defined as less than 30% of the lower limit of normative values for the ERG lab performing the test), with prolonged implicit time OU (i.e., ensure bilateral involvement) and greater rod than cone loss. If genotyping results from a certified genetic testing lab document mutations clearly diagnostic of RP, with no other potential diagnosis of a disease(s) distinct from RP, the genetic test may be used in lieu of an ERG.
- Subject age ≥ 18 years and ≤ 60 years at time of signing of consent.
- Interocular BCVA disparity ≤ 15 letters.
- Central subfield thickness (CST) ≥ 130 µm in the study eye.
- BCVA no better than 55 letters and no worse than 1 letter using the Early Treatment Diabetic Retinopathy Study (ETDRS) testing protocol in the study eye.
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- Ability to reliably fixate with the study eye at least 75% of the time as indicated by a fixation score of four (4) or five (5) on semi-automated kinetic visual fields.
- Ability to record at least two reliable trials at a minimum baseline contrast sensitivity reading of 1.28 at a minimum of one spatial frequency using the Beethoven system in the study eye.
- Central island visual field area (central island contiguous to fixation), of ≥ 50.3 deg2 (~ central island visual field diameter ≥ 8°) in the study eye.
- Willingness of subject to provide informed consent, including acknowledgement that they are able and willing to attend all required study visits, follow study protocol assessment instructions, travel by air if necessary, and provide Health Insurance Portability and Accountability Act (HIPAA) authorization.
- Willingness of subject to provide a blood sample for human leukocyte antigen (HLA) typing, if not done previously with available results.
- Willingness of subject to consent to testing for RP gene mutation typing, if not performed previously with available results.
- Adequate organ function.
- Negative active infectious disease screen (active infection with Hepatitis B, C, human immunodeficiency virus [HIV]).
- A female subject of childbearing potential must have a negative pregnancy test (urine human chorionic gonadotropin) at entry (prior to treatment).
- Women of childbearing potential must agree to use a medically accepted method of contraception for at least 12 months after jCell injection.
- For male patients whose partners are of child-bearing potential, willingness to use a medically accepted method of contraception.
Who cannot join
- Participation in any clinical trial of a drug intervention within the last 6 months, with the exception of a N-acetyl cysteine (NAC) study.
- History of ocular treatment with any non-approved, experimental, investigational or neuroprotectant therapy (systemic, topical, intravitreal) or device in either eye, including previous jCyte clinical trials. Individuals with a history of NAC treatment may take part in the study following a 7-day washout period (prior to Baseline testing).
- Subject is currently breast feeding/pumping or is planning to breast feed/pump during the 12 months after study treatment.
- Subject is pregnant or intends to become pregnant less than 12 months after jCell injection.
- Known allergy to gentamicin.
- History of adverse reaction to dimethyl sulfoxide (DMSO).
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- Prior ocular treatment with corticosteroids (systemic, periocular, intracanalicular or intravitreal - in either eye) within six months of study randomization or the anticipated need for the use of these agents to treat a pre-existing ocular condition.
- Clinical evidence of history of any eye disease or pathology, other than RP, IN EITHER EYE, that is associated with increased risk of pathology developing in the study eye, that could impair visual function, testing procedures, clinical trial endpoint measurements and/or the outcome of the study. Examples include central serous retinopathy, vitreomacular traction, pattern/vitelliform dystrophy
- Clinical evidence of history of any eye disease or pathology, other than RP, IN THE STUDY EYE, that could potentially impair visual function, testing procedures, clinical trial endpoint measurements and/or the outcome of the study.
- Concurrent use of any prohibited therapies.
- History of prior use of the following medications is prohibited if any retinal/retinal pigment epithelium (RPE) abnormalities are noted in the macula on exam or OCT: Hydroxychloroquine or chloroquine (Plaquenil); Pentosan polysulfate sodium [PPS] (Elmiron); and Interferon (Intron A, Roferon-A, IFN-alpha, alpha interferon).
- Any mental health issue likely to prevent subject from reliably performing study testing and/or examinations including dementia, schizophrenia, bipolar disorders if not reliably controlled on medications, depression if any history of hospitalization or in-patient treatment or if not sufficiently controlled on medications to enable, in the opinion of the investigator, travel to and compliance with study testing requirements over the study period.
- Uncontrolled blood pressure defined as systolic pressure > 180mmHg and/or diastolic blood pressure > 110mmHg, while subject is at rest.
- Any chronic systemic disease requiring continuous treatment with systemic steroids or immunosuppressive agents.
- History of any disease interfering with the participation in the study according to the investigator judgment, including of any type of cancer that is not in remission or considered cured, diabetes mellitus (history of gestational diabetes is acceptable), renal failure, stroke, transient ischemic attack (TIA), any systemic immune condition, any coagulopathy disorder that is not adequately managed/controlled.
- Current systemic treatment for a confirmed active infection.
- For male patients whose co-partners are of child-bearing potential, lack of willingness to use a medically accepted method of contraception, not including the rhythm method, for at least 12 months after jCell injection.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
California Retina Consultants
Bakersfield, California, 93309
Retina Consultants of Texas: Bellaire Retina Center
Bellaire, Texas, 77401
Retina-Vitreous Associates Medical Group
Beverly Hills, California, 90074
The Retina Group of Washington
Fairfax, Virginia, 22031
Vitreo Retinal Associates
Gainesville, Florida, 32607
Gavin Herbert Eye Institute, UC Irvine
Irvine, California, 92697
Illinois Retina Associates
Oak Park, Illinois, 60304
Associated Retina Consultants
Phoenix, Arizona, 85020
Retina Consultants Medical Group
Sacramento, California, 95825
Retina Consultants of Texas: San Antonio
San Antonio, Texas, 78240
NJRetina
Teaneck, New Jersey, 07666
Long Island Vitreoretinal Consultants
Westbury, New York, 11590
About this study
Official title: A Randomized, Masked, Sham-Controlled Phase 2 Trial of the Safety of a Single Intravitreal Injection of jCell (Famzeretcel) for the Treatment of Retinitis Pigmentosa (RP)
This study evaluates the safety of a single injection of jCell (famzeretcel) comprising 6.0 million (6.0M) retinal progenitor cells over a six-month study period in a cohort of adult subjects with RP. Additionally, changes in visual function will be evaluated at six months between the active treatment group (6.0M jCell) compared to sham-treated controls.
- Conditions
- Retinitis Pigmentosa
- Being tested
- human retinal progenitor cells, Mock injection
- 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, famzeretcel
Single intravitreal injection of 6.0 million retinal progenitor cells (RPCs)
Mock injectionOther
Pressing the hub of a syringe with no needle against the eye to mimic intravitreal injection. Subjects randomized to the sham control group will undergo identical preparation as the active treatment group immediately prior to treatment, including application of anesthetics.
| Study group | Role | What they receive |
|---|---|---|
| 6.0M jCell injection | Experimental | Single intravitreal injection of 6.0 million retinal progenitor cells into the study eye Receives: Biological: human retinal progenitor cells |
| Sham-treated control | Sham comparator | A mock injection will be performed on the study eye in each control subject Receives: Other: Mock injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of Intravitreal Injection of 6.0M jCell
Assessed by treatment emergent adverse events, immunogenicity and safety visual assessments
Measured: 6 months
Secondary outcomes (9)
BCVA responder rate (≥ 15 letters)
proportion of responders who achieve at least 15 ETDRS letter improvement in best corrected visual acuity (BCVA) from baseline to 6-months post-treatment
Measured: 6 months
BCVA responder rate (≥ 10 letters)
proportion of responders who achieve at least 10 ETDRS letter improvement in best corrected visual acuity (BCVA) from baseline to 6-months post-treatment
Measured: 6 months
Peak contrast sensitivity (CS) responder rate (≥ 0.3 log CS)
proportion of responders who achieve at least a 0.3 log contrast sensitivity (CS) improvement in peak CS from baseline to 6-months post-treatment
Measured: 6 months
Mean change in VA LV VFQ-48 mobility domain test scores
The VA LV VFQ-48 (VFQ) is used to capture changes in patients' self-reporting of their difficulty with reading, mobility and performing other daily living activities affected by visual impairment. There are 4 scales on the VFQ including Visual Information, Reading, Visual Motor, and Mobility. The data shown in this outcome measure is focused on the fourth scale, Mobility, and is measured in units called logits. Higher positive values on the Mobility score represent better function and less impairment, whereas lower or negatives scores represent worse function or more impairment. Change in the Mobility scale on the VFQ is calculated by taking a subject's score at 6 months and subtracting from it the baseline score.
Measured: 6 months
Mean change in VA LV VFQ-48 visual ability (overall) test scores
The VA LV VFQ-48 (VFQ) is used to capture changes in patients' self-reporting of their difficulty with reading, mobility and performing other daily living activities affected by visual impairment. There are 4 scales on the VFQ including Visual Information, Reading, Visual Motor, and Mobility. A fifth value, Visual Ability, is an aggregate score of the 4 scales, measured in units called logits, and is calculated for each person based on item weighting using Raasch analysis. Visual Ability is used broadly to represent changes in subject-reported outcomes from visit to visit. Higher positive values on the Visual Ability score represent better function and less impairment, whereas lower or negatives scores represent worse function or more impairment. Change in the Visual Ability scale on the VFQ is calculated by taking a subject's score at 6 months and subtracting from it the baseline score.
Measured: 6 months
Mean change in central island visual field area
mean change in central island visual field area (visual field that is contiguous to fixation, excluding peripheral islands), as assessed by semi-automated kinetic perimetry, from baseline to 6-months post-treatment
Measured: 6 months
Mean change in EQ-5D-5L test scores
The EQ-5D-5L is a generic measure of health status consisting of two parts. The first part (the descriptive system) assesses health in five dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) and provides a descriptive profile that can be used to generate a health state profile. Each health state can be assigned a summary index score, ranging from less than 0 (where 0 is the value of a health state equivalent to dead; negative values representing values as worse than dead) to 1 (the value of full health), with higher scores indicating higher health utility. The second part of the questionnaire consists of a visual analogue scale (VAS) on which the patient rates his/her perceived health from 0 (the worst imaginable health) to 100 (the best imaginable health). Changes in health state index and EQ VAS scores are calculated by taking a subject's scores at 6 months and subtracting from them the baseline scores.
Measured: 6 months
Mean change in peak contrast sensitivity (CS)
mean change in peak CS from baseline to 6-months post-treatment
Measured: 6 months
Mean change in BCVA
mean change in best corrected visual acuity (BCVA) from baseline to 6-months post-treatment
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Apr 2025
- Study start20 Jun 2025
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Outcomes assessor
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- jCyte, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Henry Klassen, MD, PhD, Principal investigator, jCyte, Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06912633
- Sponsor's protocol ID
- JC02-88
- Status last verified
- Jul 2026
- First submitted
- 26 Mar 2025
- Last updated
- 30 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Masked, Sham-Controlled Phase 2 Trial of the Safety of a Single Intravitreal Injection of jCell (Famzeretcel) for the Treatment of Retinitis Pigmentosa (RP)
- ClinicalTrials.gov ID
- NCT06912633
- Acronym / sponsor ID
- JC02-88
- Conditions
- Retinitis Pigmentosa
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 31 participants (actual)
- Lead sponsor
- jCyte, IncIndustry (pharma, biotech, devices)
- Study start
- 20 Jun 2025 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 6 Apr 2025 (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.
This study evaluates the safety of a single injection of jCell (famzeretcel) comprising 6.0 million (6.0M) retinal progenitor cells over a six-month study period in a cohort of adult subjects with RP. Additionally, changes in visual function will be evaluated at six months between the active treatment group (6.0M jCell) compared to sham-treated controls.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 6.0M jCell injection | Experimental | Biological: human retinal progenitor cellsSingle intravitreal injection of 6.0 million retinal progenitor cells into the study eye |
| Sham-treated control | Sham comparator | Other: Mock injectionA mock injection will be performed on the study eye in each control subject |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (17 listed)
- Clinical diagnosis of RP supported by at least 2 of the following clinical findings: (1) Loss of peripheral vision on formal visual field testing, (2) Symptoms of night blindness or difficulty adjusting to dim light, or (3) Optical coherence tomography (OCT) outer retinal atrophy consistent with RP.
- Electroretinography (ERG) results that support diagnosis of RP including nondetectable or severely reduced rod responses (defined as less than 30% of the lower limit of normative values for the ERG lab performing the test), with prolonged implicit time OU (i.e., ensure bilateral involvement) and greater rod than cone loss. If genotyping results from a certified genetic testing lab document mutations clearly diagnostic of RP, with no other potential diagnosis of a disease(s) distinct from RP, the genetic test may be used in lieu of an ERG.
- Subject age ≥ 18 years and ≤ 60 years at time of signing of consent.
- Interocular BCVA disparity ≤ 15 letters.
- Central subfield thickness (CST) ≥ 130 µm in the study eye.
Key exclusion criteria (17 listed)
- Participation in any clinical trial of a drug intervention within the last 6 months, with the exception of a N-acetyl cysteine (NAC) study.
- History of ocular treatment with any non-approved, experimental, investigational or neuroprotectant therapy (systemic, topical, intravitreal) or device in either eye, including previous jCyte clinical trials. Individuals with a history of NAC treatment may take part in the study following a 7-day washout period (prior to Baseline testing).
- Subject is currently breast feeding/pumping or is planning to breast feed/pump during the 12 months after study treatment.
- Subject is pregnant or intends to become pregnant less than 12 months after jCell injection.
- Known allergy to gentamicin.
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: jCell, famzeretcelSingle intravitreal injection of 6.0 million retinal progenitor cells (RPCs) | Biological | 6.0M jCell injection |
| Mock injectionPressing the hub of a syringe with no needle against the eye to mimic intravitreal injection. Subjects randomized to the sham control group will undergo identical preparation as the active treatment group immediately prior to treatment, including application of anesthetics. | Other | Sham-treated control |
Primary outcome
Safety of Intravitreal Injection of 6.0M jCell
Time frame: 6 months
Assessed by treatment emergent adverse events, immunogenicity and safety visual assessments
9 secondary outcomes
BCVA responder rate (≥ 15 letters)
Time frame: 6 months
BCVA responder rate (≥ 10 letters)
Time frame: 6 months
Peak contrast sensitivity (CS) responder rate (≥ 0.3 log CS)
Time frame: 6 months
Mean change in VA LV VFQ-48 mobility domain test scores
Time frame: 6 months
Mean change in VA LV VFQ-48 visual ability (overall) test scores
Time frame: 6 months
Mean change in central island visual field area
Time frame: 6 months
Mean change in EQ-5D-5L test scores
Time frame: 6 months
Mean change in peak contrast sensitivity (CS)
Time frame: 6 months
Mean change in BCVA
Time frame: 6 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 30 Jul 2026.
- JC02-88 Study WebsiteLink provided by the sponsor.
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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. This study is not recruiting at the moment.
Henry Klassen, MD, PhD principal investigator
jCyte, Inc
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing human retinal progenitor cells and Mock injection in people with Retinitis Pigmentosa, run by jCyte, Inc at 12 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 17 inclusion and 17 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: under way, not looking for participants, as of the sponsor's last update on 30 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
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?
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?
Safety of Intravitreal Injection of 6.0M jCell (time frame: 6 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 30 Jul 2026.