Long Term Follow Up study of Gene Therapy Trial for Patients with RPGR
Research type
Research Study
Full title
Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Protocol MGT-RPGR-022
IRAS ID
287029
Contact name
James Bainbridge
Contact email
Sponsor organisation
MeiraGTx UK II Limited
Eudract number
2020-002255-37
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
EDMS-RIM-34175, 1.0, EDMS number
Duration of Study in the UK
5 years, 9 months, 0 days
Research summary
This MGT‐RPGR‐022 study is a follow‐up study to MGT‐RPGR‐021.
After participants have completed the first study (MGT‐RPGR‐021), they will be invited to continue to the second study (MGT‐RPGR‐022). Participants who did not receive the intervention in MGT‐RPGR‐021 will receive the intervention in MGT‐RPGR‐022, in both eyes.
The purpose of this study is to see if the investigational gene therapy is safe and useful in patients not treated in the MGT‐RPGR‐021 study
and to extend follow‐up for patients that received intervention in the MGT‐RPGR‐021 or MGTRPGR‐022 studies.
The total duration of this study will be for a period of 4 to 5 years. The length of participation will depend on participants' intervention assignment from MGT‐RPGR‐021. Participants will need to
review the information for both studies and decide whether they agree to participate.
Participants treated in MGT‐RPGR‐021 will be followed for 4 years and participants not treated in MGT‐RPGR‐021 will receive treatment in MGTRPGR‐022 according to the same schedule as participants treated in 021 and then followed for an additional 4 years.
During both studies, the sponsor may learn new information about the investigational gene therapy, the risks, and additional information.
Participants' doctor/staff will tell participants in a timely manner if there is any new information that might make participants change their mind about being in the studies.REC name
London - West London & GTAC Research Ethics Committee
REC reference
21/LO/0046
Date of REC Opinion
18 May 2021
REC opinion
Further Information Favourable Opinion