LONG TERM FOLLOW-UP GENE THERAPY STUDY FOR XLRP RPGR
Research type
Research Study
Full title
Long term follow-up study of participants following an open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-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
247578
Contact name
James Bainbridge
Contact email
Sponsor organisation
MeiraGTx UKII Ltd
Eudract number
2018-000425-31
Duration of Study in the UK
5 years, 7 months, 30 days
Research summary
The follow up study is designed to collect data on longer team safety and efficacy at 24, 36, 48 and 60 months following AAV2/5-hRKp.RPGR ATIMP administration in the RPGR trial. The RPGR trial is an on-going open-label, Phase I/II dose-escalation study to determine the safety and efficacy of a single subretinal administration of the ATIMP in participants with RPGR-related retinal dystrophy.
In the dose escalation phase, up to 18 adult participants are administered one of 3 different doses of vector in cohorts of 3 participants at a time, using a 3+3 design. Based on toxicity data, the IDMC makes a recommendation on the dose to administer to the next cohort of 3 participants. The IDMC may recommend expanding cohorts on safety grounds.
Up to 18 children or adults will be included once an acceptable safety profile has been established in adults. The IDMC will agree the maximum tolerated dose in adults before recommending administration upto this dose to children.
Safety and efficacy is being assessed by clinical examination and investigations according to a pre-defined schedule of follow up visits, for 18 months following the intervention.
At the 12 or 18 month follow up visit of the RPGR trial (12 or 18 months post administration) participants will be invited to join this longer term follow up study, in which they will be assessed for safety for up to 60 months following AAV2/5-hRKp.RPGR ATIMP administration.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
18/LO/1098
Date of REC Opinion
30 Aug 2018
REC opinion
Favourable Opinion