LONG-TERM FOLLOW-UP GENE THERAPY STUDY FOR LCA OPTIRPE65
Research type
Research Study
Full title
Long term follow-up study of participants in an open label, multi-centre, Phase I/II dose escalation trial of an adeno-associated virus vector (AAV2/5-OPTIRPE65) for gene therapy of adults and children with retinal dystrophy associated with defects in RPE65 (LCA2)
IRAS ID
205081
Contact name
Stuart Naylor
Contact email
Sponsor organisation
MeiraGTx UKII Ltd
Eudract number
2016-000898-20
Clinicaltrials.gov Identifier
EudraCT, 2015-003418-25
Duration of Study in the UK
7 years, 3 months, 1 days
Research summary
The follow up study is designed to collect data on longer-term safety and efficacy at 9, 12, 24, 36, 48 and 60 month time-points following AAV2/5-OPTIRPE65 administration in the OPTIRPE65 trial. The OPTIRPE65 trial is an on-going open-label, Phase I/II dose-escalation study to determine the safety and efficacy of a subretinal administration of the ATIMP in participants with RPE65-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 an additional 1 or 2 participants be administered any dose before making another decision.
Up to 9 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 of this dose to children.
Safety and efficacy is being assessed by clinical examination and special investigations according to a pre-defined schedule of follow up visits, for 6 months following the intervention.
At the 3 month follow up visit of the OPTIRPE65 trial (3 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-OPTIRPE65 administration.REC name
London - West London & GTAC Research Ethics Committee
REC reference
16/LO/1254
Date of REC Opinion
4 Oct 2016
REC opinion
Further Information Favourable Opinion