GS030_CLIN_001, Phase 1/2a, Open-Label study for Retinitis Pigmentosa
Research type
Research Study
Full title
A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects with Retinitis Pigmentosa
IRAS ID
235242
Contact name
Simona Esposti
Contact email
Sponsor organisation
GENSIGHT-BIOLOGICS
Eudract number
2017-002204-27
Duration of Study in the UK
5 years, 0 months, 1 days
Research summary
This is a phase 1/2a study for patients with Retinitis Pigmentosa (RP). RP is caused by the progressive loss of certain cells in the retina that allow the eyes detect visual stimuli. RP is an inherited disease, which means it is caused by deficiencies certain gene(s).
In this study, the damaged cells have defects in genes which cause photoreceptor cells (the cells of the retina that naturally perceive light) to breakdown and die. The experimental gene therapy product GS030-DP will be injected into the jelly-like area within the eyeball which is called the vitreous humour. This injection process is known as an intravitreal injection (IVT). It is very commonly used to deliver other types of medication, such as medication for macular degeneration, to the eye.
The study treatment GS030 combines the action of two components: a medicine: GS030-DP and a medical device: GS030-MD (stimulating glasses). The purpose of this study is to learn if treatment with GS030 is safe, tolerable and effective.The study is split into three sections, screening – to check for eligibility, treatment and follow up. Eligible patients will begin the treatment phase. The first three patients will be assigned the lowest dose, based on safety data obtained from them; the next patients enrolled may begin treatment at the same, or a higher dose. The treatment phase will last one day. The follow up phase will last one year with approximately 9 visits. A long term follow up visit will be conducted at 1.5, 2, 3, 4 and 5 years after GS030-DP administration.
The study will take place at one NHS hospital in the UK and one hospital in France. 9 patients are expected to be recruited in the UK and 9 in France: totalling 18 patients.
REC name
North East - York Research Ethics Committee
REC reference
17/NE/0374
Date of REC Opinion
22 Dec 2017
REC opinion
Further Information Favourable Opinion