RPG2001
Research type
Research Study
Full title
An Open-label, Multicenter, Phase 2 Follow-on Study for Second Eye Treatment of Patients Previously Treated With a Recombinant Adeno-associated Virus Vector (AAV5 hRKp.RPGR) for Gene Therapy of Adults and Children with X-linked Retinitis Pigmentosa Owing to defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
IRAS ID
1010255
Contact name
David Wright
Contact email
Sponsor organisation
Janssen-Cilag International NV
Research summary
X-linked retinitis pigmentosa (XLRP) is an inherited eye condition that causes severe eye problems, eventually leading to blindness. It is commonly caused due to an alteration in a gene* called retinitis pigmentosa guanosine triphosphatase regulator (RPGR). Currently, there are no approved treatments available for XLRP.
*A part of DNA that contains the information for making a specific protein.
The study drug, AAV5-hRKp.RPGR, is a modified gene delivery system that carries a healthy version of the RPGR gene into the cells of an eye. This gene may help to improve and preserve vision. In this study, researchers want to learn how safe and tolerable it is to deliver AAV5-hRKp.RPGRunder the retina into the second eye of a person who has already received it in their first eye.
Male participants aged 5 years & older, previously treated with AAV5-hRKp.RPGR in one eye in the study MGT009 and have completed or are currently enrolled in Study MGT010.
Participants will be divided into 3 cohorts:
• Participants who are eligible to receive AAV5-hRKp.RPGR in the second eye through surgery will be enrolled sequentially (one after the other) into 2 cohorts:
o Cohort 1: After receiving treatment, participants will be assessed for safety through Week 12.
o Cohort 2: Participants will receive the treatment once safety is determined in Cohort 1.
o Cohort 3: Participants who do not wish to undergo surgery or are not eligible for surgery.
Study consists of:
1. Screening/Baseline period (up to 6 months)
2. Administration of AAV5-hRKp.RPGR under the retina (Day 1): Participants may receive a dose depending on the dosage administered in study MGT009 in the past.
3. Assessment period (52 weeks): Participants will undergo study assessments & tests including blood tests, imaging scans, eye examinations, efficacy outcome assessments & monitoring of side effects.
4. Long-term follow-up (4 years): The overall duration of the study will be approximately 5 years and 6 months.REC name
London - West London & GTAC Research Ethics Committee
REC reference
24/LO/0470
Date of REC Opinion
1 Oct 2024
REC opinion
Further Information Favourable Opinion