A Phase I/II clinical trial of gene therapy for X-linked Retinitis Pigmentosa run by Nightstar in collaboration with Professor Robert MacLaren (Oxford team) and Professor Paulo Stanga (Manchester team).
What is the study about and what is it trying to achieve?
X-linked Retinitis Pigmentosa (XLRP) is an inherited genetic disease that results in progressive retinal degeneration, which eventually leads to blindness. The disease is caused by a defect in a certain gene located on the X-chromosome, which is why the disease affects men and women differently.
There are currently no effective treatments available for XLRP. This clinical trial will investigate the safety, tolerability and efficacy of a retinal gene therapy with an adeno-associated viral (AAV) vector encoding the defective gene Retinitis Pigmentosa GTPase Regulater (RPGR).
This study will provide important information regarding the use of retinal gene therapy in patients with XLRP.
Who can participate in the study?
- Patients diagnosed with XLRP (with RPGR mutation)
- Male and >= 18 years of age
Additional inclusion criteria are applicable depending on the results of various clinical tests. We would be happy to review potentially eligible patients in the clinic to provide further details. A written referral from the patient’s GP or hospital consultant would be required.
Study target enrolment: approximately 30 patients
Sites involved: Oxford Eye Hospital, Manchester Royal Eye Hospital
Principle Investigators: Prof Robert MacLaren.
ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT03116113
If you are interested in taking part in this or similar future studies please contact Biogen:
This phase of the trial has closed and we are awaiting phase 3 to commence. Date TBC.