CNS10-NPC for the Treatment of RP (NCT04284293) | Clinical Trial Compass
Active — Not RecruitingPhase 1
CNS10-NPC for the Treatment of RP
United States16 participantsStarted 2021-07-22
Plain-language summary
The investigator is examining the safety of transplanting cells into the subretinal space of patients with Retinitis Pigmentosa (RP). The cells are called neural progenitor cells, which are a type of stem cell that can become several different types of cells in the nervous system. These cells have been derived to specifically become astrocytes, which is a type of neuronal cell. The cells are called "CNS10-NPC." The investigational treatment has been tested in animals, but it has not yet been tested in people. In this study, the investigators want to learn if CNS10-NPC cells are safe to transplant into the subretinal space of people.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. To participate in this study, the subject must be 18 years of age or older and must understand and sign the protocol's informed consent.
. Participant with diagnosis of retinitis pigmentosa.
Exclusion criteria
. Presence of significant ocular abnormalities that would preclude the planned surgery or interfere with the interpretation of study endpoints (e.g. glaucoma, corneal or significant lens opacities, pre-existing uveitis, intraocular infection, macular edema, choroidal neovascularization). Any ocular diseases that the investigator feels would interfere with accurate ocular measurements. This would exclude potential subjects with significant cataract, corneal scars or significant corneal irregularities such as keratoconus, previous penetrating keratoplasty or glaucoma with central visual field.
. Any pre-existing factor or history of eye disease that may predispose to an increased risk of surgical complications in the study eye (e.g. trauma, previous surgery other than uncomplicated cataract surgery, uveitis, congenital developmental or structural abnormalities).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Safety as evaluated by incidence of Adverse Events (AE) and Serious Adverse Events (SAE) and their relationship to the intervention
Timeframe: Subjects will be followed postoperatively for 12 months
2
Safety, as evaluated by changes in the complete blood count
Timeframe: Subjects will be followed postoperatively for 12 months
3
Safety, as evaluated by changes in the comprehensive metabolic panel
Timeframe: Subjects will be followed postoperatively for 12 months
4
Safety, as evaluated by changes in Donor Specific Antibodies
Timeframe: Subjects will be followed postoperatively for 12 months
5
Safety, as evaluated by changes in the urinalysis
Timeframe: Subjects will be followed postoperatively for 12 months
6
Safety, as evaluated by changes in Visual Acuity
Timeframe: Subjects will be followed postoperatively for 12 months
. Concomitant systemic diseases including those in which the disease itself, or the treatment for the disease, can alter ocular function or immune status (e.g. malignancies, uncontrolled diabetes).
. Any ocular surgery or laser in either eye within 12 weeks of screening.
. Any contraindication to pupil dilatation in either eye.
. Treatment with intravitreal, subtenon or periocular steroid within 4 months of enrollment.
. Any known allergy to any component of the delivery vehicle or diagnostic agents used during the study (e.g., dilation drops), or medications planned for use during the peri-operative period including corticosteroids, tacrolimus and mycophenolate.
. Imminently life-threatening illness.
Safety, as evaluated by changes in visual field
Timeframe: Subjects will be followed postoperatively for 12 months
8
Safety, as evaluated by changes in Spectral Domain Optical Coherence Tomography (SD-OCT)
Timeframe: Subjects will be followed postoperatively for 12 months