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Tests treatment safety and results for Retinitis Pigmentosa
Official title Safety of a Single, Intravitreal Injection of 6.0M jCell (Famzeretcel) in Retinitis Pigmentosa (RP)
ClinicalTrials.gov ID: NCT06912633
What this study is testing
What is human retinal progenitor cells?
human retinal progenitor cells is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for retinitis pigmentosa.
Also referred to as jCell, famzeretcel.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 60
You may be able to join if
- Clinical diagnosis of RP supported by at least 2 of the following clinical findings: (1) Loss of peripheral vision on formal visual field testing...
- Electroretinography (ERG) results that support diagnosis of RP including nondetectable or severely reduced rod responses (defined as less than 30% of...
- 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.
You likely can't join if
- 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...
- 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).
See the full eligibility criteria
- 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...
- 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...
- 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.
- 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...
- 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.
- 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...
- 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).
- 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...
- 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...
- 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...
- 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)...
- 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...
- 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Phoenix, Arizona, United States
- Bakersfield, California, United States
- Beverly Hills, California, United States
- Irvine, California, United States
- Sacramento, California, United States
- Walnut Creek, California, United States
- Gainesville, Florida, United States
- Marietta, Georgia, United States
- Oak Park, Illinois, United States
- Teaneck, New Jersey, United States
- Westbury, New York, United States
- Bellaire, Texas, United States
- San Antonio, Texas, United States
- Fairfax, Virginia, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 60 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Phoenix, Arizona, United States; Bakersfield, California, United States; Beverly Hills, California, United States; Irvine, California, United States; Sacramento, California, United States; Walnut Creek, California, United States and 8 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.