Safety & tolerability of hRPC in patients with Retinitis Pigmentosa
Research type
Research Study
Full title
First-in-human Phase I/IIa, Open-Label, Prospective Study of the Safety and Tolerability of Subretinally Transplanted Human Retinal Progenitor Cells (hRPC) in Patients with Retinitis Pigmentosa (RP)
IRAS ID
279052
Contact name
Vincent Holmes
Contact email
Sponsor organisation
ReNeuron
Eudract number
2019-004547-77
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 3 months, 30 days
Research summary
RP is a genetic form of human blindness due to degeneration of the retina and there is currently no approved drug treatment for the vast majority of RP sufferers.This study aims to identify the safety and tolerability of (human retinal progenitor cells-hRPC) when implanted into patients with RP. The sponsor, ReNeuron Ltd, has manufactured a cell therapy that when implanted into the retina of the eye can preserve vision in animals that have blindness. This study aims to evaluate four doses of these cells with the purpose of identifying the highest tolerable and safe dose in patients with advanced retinitis pigmentosa. Patients will be followed-up closely for up to 24 months over 17 visits. They will under a series of eye examinations and general health examinations to make sure they are suitable for the study.
The study is open-label, so all participants will receive hRPC and be aware of what dose they have received.
The Phase I portion of the study is now complete and 4 cohorts of phase IIa are complete. The study will dose up to 9 subjects at 2 million cells over 3 cohorts. There will be a safety review after at least the 1st and 3rd patient dosed at this new level before proceeding to patients 4-9.
Participants meeting the eligibility criteria and having signed informed consent will be enrolled. All participants will receive treatment consisting of two or more injections of stems cell into the eye through a surgical procedure call a vitrectomy. The eye with the worst vision will be enrolled as the study eye. The second, untreated eye will be assessed as the control eye. Where no difference between the eligible eyes the non-dominant eye will be selected.REC name
London - West London & GTAC Research Ethics Committee
REC reference
20/LO/0564
Date of REC Opinion
9 Jun 2020
REC opinion
Further Information Favourable Opinion