jCell for Retinitis Pigmentosa

Recruiting at 2 trial locations

Overseen By jCyte Sr. Director of Clinical Operations

Age: 18 - 65

Sex: Any

Trial Phase: Phase 2

Sponsor: jCyte, Inc

Must not be taking: Corticosteroids, Hydroxychloroquine, Interferon, others

Disqualifiers: Pregnancy, Breastfeeding, Uncontrolled hypertension, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 1

Duration: 1 day

60 Participants Needed

Trial Summary

What is the purpose of this trial?

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 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 (8.8M jCell) compared to sham-treated controls.

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.

What data supports the effectiveness of the treatment jCell (Famzeretcel) for Retinitis Pigmentosa?

The research highlights that jCell is one of the leading cell therapies in late-stage development for Retinitis Pigmentosa, suggesting its potential effectiveness. Cell therapies like jCell are noted for their broader application as they do not rely on the presence of viable photoreceptors, which may make them suitable for treating hereditary retinal diseases like Retinitis Pigmentosa. ^{1 2 3 4 5}

Is jCell (Famzeretcel) safe for humans?

In a study involving 18 patients with macular degeneration, a similar stem cell-derived treatment was safely implanted without major adverse events like tumor growth or rejection-related inflammation, suggesting that such treatments can be generally safe in humans. ^{6 7 8 9 10}

What makes the jCell treatment for retinitis pigmentosa unique?

The jCell treatment, also known as Famzeretcel, is unique because it involves the use of induced pluripotent stem cells (iPSCs) to address the underlying genetic causes of retinitis pigmentosa, a condition with no standard treatment. This approach aims to restore the function of retinal cells by potentially correcting genetic defects, which is different from traditional treatments that mainly focus on managing symptoms. ^{7 11 12 13 14}

Research Team

Henry Klassen, MD, PhD

Principal Investigator

jCyte, Inc

Eligibility Criteria

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

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.

Timeline

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)

Treatment Details

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.

Participant Groups

Group I: 8.8M jCell injection Experimental Treatment 1 Intervention

Single intravitreal injection of 8.8 million retinal progenitor cells into the study eye

Group II: Sham-treated control Placebo Group 1 Intervention

A mock injection will be performed on the study eye in each control subject

Who Is Running the Clinical Trial?

jCyte, Inc

Lead Sponsor

Trials

3

Recruited

140+

Findings from Research

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Current and Future Treatment of Retinitis Pigmentosa. Cross, N., van Steen, C., Zegaoui, Y., 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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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]

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The first gene therapy for RPE65 biallelic dystrophy with voretigene neparvovec-rzyl in Brazil. Ferraz Sallum, JM., Godoy, J., Kondo, A., et al.[2022]

References

1. England pubmed.ncbi.nlm.nih.gov

Photoreceptor integrity and visual acuity in cystoid macular oedema associated with retinitis pigmentosa. [2022]

2. New Zealand pubmed.ncbi.nlm.nih.gov

Current and Future Treatment of Retinitis Pigmentosa. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

A RANDOMIZED PAIRED-EYE TRIAL OF INTRAVITREAL DEXAMETHASONE IMPLANT FOR CYSTOID MACULAR EDEMA IN RETINITIS PIGMENTOSA. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Autosomal dominantly inherited retinitis pigmentosa. Visual acuity loss by subtype. [2019]

5. United States pubmed.ncbi.nlm.nih.gov

Pathophysiology and treatment of clinically resistant cytomegalovirus retinitis. [2019]

6. Switzerland pubmed.ncbi.nlm.nih.gov

Retinal Pigment Epithelium Remodeling in Mouse Models of Retinitis Pigmentosa. [2021]

7. England pubmed.ncbi.nlm.nih.gov

Human iPSC derived disease model of MERTK-associated retinitis pigmentosa. [2018]

8. United States pubmed.ncbi.nlm.nih.gov

Subretinal Transplantation of Embryonic Stem Cell-Derived Retinal Pigment Epithelium for the Treatment of Macular Degeneration: An Assessment at 4 Years. [2022]

9. United States pubmed.ncbi.nlm.nih.gov

Inflammation of the retinal pigment epithelium drives early-onset photoreceptor degeneration in Mertk-associated retinitis pigmentosa. [2023]

10. France pubmed.ncbi.nlm.nih.gov

[Gene therapy for retinitis pigmentosa]. [2022]

11. England pubmed.ncbi.nlm.nih.gov

Deterioration of phagocytosis in induced pluripotent stem cell-derived retinal pigment epithelial cells established from patients with retinitis pigmentosa carrying Mer tyrosine kinase mutations. [2021]

12. England pubmed.ncbi.nlm.nih.gov

Generation of two induced pluripotent stem cell lines from a patient with dominant PRPF31 mutation and a related non-penetrant carrier. [2021]

13. England pubmed.ncbi.nlm.nih.gov

Using inducible lentiviral vectors to generate induced pluripotent stem cell line ZOCi001-A from peripheral blood cells of a patient with CRB1-/- retinitis pigmentosa. [2021]

14. England pubmed.ncbi.nlm.nih.gov

The first gene therapy for RPE65 biallelic dystrophy with voretigene neparvovec-rzyl in Brazil. [2022]