VISTA
Research type
Research Study
Full title
A Randomized, Controlled, Masked, Multi-center Study Evaluating the Efficacy, Safety, and Tolerability of Two Doses of AGTC-501 Compared to an Untreated Control Group in Male Participants with X-linked Retinitis Pigmentosa
IRAS ID
1009737
Contact name
Tricia Racanelli
Contact email
Sponsor organisation
Beacon Therapeutics
Eudract number
2024-511181-36
Clinicaltrials.gov Identifier
Research summary
Retinitis pigmentosa (RP) is the name for rare genetic eye diseases causing the light-sensitive tissue at the back of the eye (retina) to break down over time leading to loss of vision. About 10% of RP cases are a disease known as X-Linked Retinitis Pigmentosa (XLRP). XLRP is caused by a genetic mutation in a gene called RPGR and mostly affects boys and young men. The symptoms of XLRP start with night blindness, followed by a gradual loss of vision. It may cause patients to become blind by age 40. Currently there is no cure for XLRP.
Beacon Therapeutics is developing an experimental drug called AGTC-501, to improve vision and symptoms in patients with XLRP. This study drug contains healthy copies of the RPGR gene and parts of a small virus (called adeno-associated virus or AAV). By injecting the study drug into the eye, the AAV works as a delivery system to get healthy gene copies into the patient’s retina cells where they will replace the mutated gene that causes XLRP. This experimental procedure is called gene transfer.
This study will evaluate the safety, effectiveness and tolerability of a single injection of AGTC-501 (in one of two doses) into the eye, compared to an untreated group. Patients will be assigned randomly to either receive the experimental drug or be in the untreated group (see groups below). Approximately 75 male XLRP patients between 12 and 50 years of age will be enrolled in this study. The study will last for up to 6 years.
• Group 1: high-dose treatment group
• Group 2: low-dose treatment group
• Group 3: untreated group (these participants may receive the study drug after Month 12)REC name
London - West London & GTAC Research Ethics Committee
REC reference
24/LO/0310
Date of REC Opinion
30 May 2024
REC opinion
Further Information Favourable Opinion