hRPC is a cell therapy for retinitis pigmentosa. This is a first-in-human, dose escalation
study in which participants with retinitis pigmentosa will receive a single subretinal
injection of hRPC cells in one eye to evaluate safety and tolerability.
Participants will be followed for one year to evaluate the safety and tolerability of hRPC
Additional testing will seek to establish any preliminary efficacy from hRPC.
This is a first-in-human open label phase I/II dose-escalation study in which participants
with retinitis pigmentosa will receive a single uni-ocular subretinal implantation of one of
three doses of hRPC.
Treated eyes will be carefully monitored for any ocular or systemic adverse events for 1
Testing will comprise a series of detailed ophthalmic examinations and imaging together with
blood testing and systemic evaluations, as necessary Ophthalmic testing will also be
evaluated for any preliminary efficacy signal.
- Adult male or female over 18 years of age.
- Clinical diagnosis of RP, based upon one or more of the following: clinical features,
electrophysiological measures and genetic testing, if available (genetic confirmation
is not obligatory).
- Best corrected Visual acuity of 20/200 or worse in the study eye (N.B. in patients
with differing acuities between eyes, the worse eye will be enrolled as the study
- Medically able to undergo vitrectomy and subretinal injection, a surgery which may
require general anesthesia.
- Good general health as defined by:
- Normal serum chemistry and hematology. Out of normal range laboratory findings deemed
not clinical significant are acceptable.
- No history of malignancy, except non-melanoma skin cancer; pre-malignant conditions
and cancer in situ.
- Negative serology for human immunodeficiency virus (HIV), hepatitis B (HBV), hepatitis
- Female patients of child-bearing potential must use a reliable method of contraception
(e.g. oral contraceptive and condom, intra-uterine device (IUD) and condom, diaphragm
with spermicide and condom) during the study.
- Females of childbearing potential must have a confirmed negative pregnancy test at
time of treatment.
- Males must be willing to use barrier and spermicide contraception during the study.
- Ability to give written informed consent as evidenced by signature on the patient
consent form, to communicate well with the investigator and to comply with the
expectations of the study.
- The presence of ocular disease or ocular media opacity, which in the opinion of the
investigator, precludes accurate evaluation during the study.
- Prior vitrectomy in the study eye
- Patients with a history of amblyopia will be excluded
- High myopia (>6 diopters) in the study eye
- Cataract surgery in the study within 3 months
- Participation within 6 months in any clinical trial involving a drug or device
- No prior stem cell injections in any part of the body
- Use of systemic immunosuppressive agents in the six months prior to treatment