60 Participants Needed

jCell for Retinitis Pigmentosa

Recruiting at 5 trial locations
jS
Overseen ByjCyte Sr. Director of Clinical Operations
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called jCell for individuals with retinitis pigmentosa (RP), a condition that impairs vision, particularly in low light. Researchers aim to determine if a single injection of jCell can safely enhance vision over six months. Participants will receive either the jCell treatment or a sham (fake) injection for comparison. Suitable candidates have been diagnosed with RP and experience symptoms such as loss of peripheral vision or difficulty seeing in dim light. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group, providing participants an opportunity to contribute to significant findings.

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop taking your current medications. However, you cannot participate if you are using certain prohibited therapies or if you have used specific medications like hydroxychloroquine, chloroquine, or interferon with noted retinal abnormalities. If you were part of a N-acetyl cysteine (NAC) study, a 7-day washout period is required.

Is there any evidence suggesting that jCell (Famzeretcel) is likely to be safe for humans?

Research has shown that jCell (famzeretcel) is safe and well-tolerated. Studies found that a single injection of these eye cells did not cause any serious safety issues, and participants did not reject the cells. Most side effects were mild to moderate and resolved on their own. This suggests the treatment is generally safe for humans.12345

Why do researchers think this study treatment might be promising for retinitis pigmentosa?

Unlike the standard treatments for retinitis pigmentosa, which mainly focus on managing symptoms and slowing disease progression, jCell (Famzeretcel) offers a regenerative approach. This treatment is unique because it involves a single injection of 8.8 million retinal progenitor cells directly into the eye, potentially replenishing damaged cells and restoring vision. Researchers are excited about jCell because it targets the underlying cause of retinal degeneration, which could lead to significant improvements in vision, offering hope for a condition that currently has no cure.

What evidence suggests that jCell might be an effective treatment for retinitis pigmentosa?

Research shows that jCell, also known as famzeretcel, may improve vision in people with retinitis pigmentosa (RP). In earlier studies, patients who received one injection of jCell showed possible improvements in vision sharpness compared to those who did not receive it. Some studies indicate that these improvements might be more noticeable with higher doses. In this trial, participants will receive either an 8.8 million jCell injection or a sham-treated control. The treatment proved to be safe and well-tolerated, with no serious side effects reported. Although the results are still early, they suggest that jCell might help some people with RP see better.14567

Who Is on the Research Team?

HK

Henry Klassen, MD, PhD

Principal Investigator

jCyte, Inc

Are You a Good Fit for This Trial?

This trial is for adults with Retinitis Pigmentosa (RP), a genetic eye condition that leads to vision loss. Participants must be eligible based on specific health criteria not detailed here.

Inclusion Criteria

Electroretinography (ERG) results supporting diagnosis of RP including nondetectable or severely reduced rod responses, prolonged implicit time OU, greater rod than cone loss
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
Interocular BCVA disparity ≤ 15 letters
See 13 more

Exclusion Criteria

Subject is currently breast feeding/pumping or planning to breast feed/pump during the 12 months after study treatment
I have used eye steroids in the last 6 months or might need them.
I've used specific medications and have abnormal retinal findings.
See 14 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravitreal injection of 8.8 million retinal progenitor cells or a sham injection

1 day
1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with assessments of visual function

6 months
Multiple visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

  • jCell (Famzeretcel)
Trial Overview The study tests the safety of an injection called jCell, which contains 8.8 million retinal progenitor cells, compared to a mock injection in people with RP over six months.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Placebo Group
Group I: 8.8M jCell injectionExperimental Treatment1 Intervention
Group II: Sham-treated controlPlacebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

jCyte, Inc

Lead Sponsor

Trials
3
Recruited
140+

Published Research Related to This Trial

There are over 100 drugs currently in development for Retinitis Pigmentosa (RP), with 50% being advanced therapy medicinal products (ATMPs), highlighting a significant push towards innovative treatments for this condition.
Gene therapies aim to restore vision by targeting non-functional photoreceptors, while cell therapies offer broader applications as they do not depend on the presence of viable photoreceptors, making them potentially more accessible for patients at various disease stages.
Current and Future Treatment of Retinitis Pigmentosa.Cross, N., van Steen, C., Zegaoui, Y., et al.[2022]
The first Brazilian patient with RPE65 deficiency was treated with voretigene neparvovec-rzyl (VN), showing no complications and stable best-corrected visual acuity after treatment.
Significant improvements were observed in the patient's full-field stimulus threshold and visual fields, enhancing their ability to perform daily activities, particularly in low-light conditions.
The first gene therapy for RPE65 biallelic dystrophy with voretigene neparvovec-rzyl in Brazil.Ferraz Sallum, JM., Godoy, J., Kondo, A., et al.[2022]
Two phase I/II studies involving 18 patients with advanced macular degeneration showed that implanting human embryonic stem cell-derived retinal pigment epithelium (hESC-RPE) is safe and may improve visual acuity in over half of the treated patients.
No significant adverse events related to the cell therapy were observed, aside from one case of postoperative infection, suggesting a favorable safety profile for future studies and potential treatments for other retinal diseases.
Subretinal Transplantation of Embryonic Stem Cell-Derived Retinal Pigment Epithelium for the Treatment of Macular Degeneration: An Assessment at 4 Years.Schwartz, SD., Tan, G., Hosseini, H., et al.[2022]

Citations

jCyte Announces Publication of Phase I/IIa Safety Study ...“These encouraging results demonstrate that a single intravitreal injection of RPCs was safe and well tolerated, with exploratory signals ...
2.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/40927136/
Retinal progenitor cells (jCell) for retinitis pigmentosaThe exploratory efficacy data suggest potential improvement of BCVA in some RP patients, particularly at the highest dose. While viewed ...
NCT03073733 | Safety and Efficacy of Intravitreal Injection ...This study evaluates the changes in visual function at 12 months following a single injection of human retinal progenitor cells compared to sham treated ...
Retinal progenitor cells (jCell) for retinitis pigmentosaWhile outcomes from this small safety study are preliminary, the data are suggestive of an improvement of visual acuity in some patients with this ...
JC02-88 Study PatientsThe study also aims to evaluate the potential effectiveness of jCell in improving vision and quality of life over a 6-month period. The study is expected to ...
jCyte publishes Phase I/IIa safety study resultsNo one experienced serious safety problems, and the cells did not cause rejection.
Safety of a Single, Intravitreal Injection of 8.8M jCell ...This study evaluates the safety of a single injection of jCell (famzeretcel) comprising 8.8 million (8.8M) retinal progenitor cells over a six-month study ...
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