NCT02464436

Safety and Tolerability of hRPC in Retinitis Pigmentosa

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)

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 29 (actual)
• Sponsor: ReNeuron Limited · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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 two years to evaluate the safety and tolerability of hRPC Additional testing will seek to establish any preliminary efficacy from hRPC.

Detailed description

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 2 years. 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.

Conditions

• Retinitis Pigmentosa

Interventions

Type	Name	Description
DRUG	hRPC	Participants will undergo vitrectomy surgery and subretinal implantation of hRPC in the study eye.

Timeline

• Start date: 2015-12-01
• Primary completion: 2022-06-01
• Completion: 2023-12-01
• First posted: 2015-06-08
• Last updated: 2023-07-06

Locations

5 sites across 3 countries: United States, Spain, United Kingdom