Long-Term Follow-Up Gene Therapy Study for Achromatopsia (CNGB3 and CNGA3)
Research type
Research Study
Full title
Long-term follow-up study of participants following open label, multicentre, Phase I/II dose escalation trials of a recombinant adeno-associated virus vector (AAV8-hCARp.hCNGB3 or AAV8-hG1.7p.coCNGA3) for gene therapy of adults and children with achromatopsia owing to defects in CNGB3 or CNGA3
IRAS ID
216171
Contact name
James Bainbridge
Contact email
Sponsor organisation
MeiraGTx UK II Ltd
Eudract number
2016-003856-59
Duration of Study in the UK
6 years, 2 months, 22 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 AAV2/8-hCARp.hCNGB3 administration in the CNGB3 trial. The Achromatopsia CNGB3 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 achromatopsia.
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 additional participant are 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 CNGB3 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 AAV2/8-hCARp.hCNGB3 administration.REC name
London - West London & GTAC Research Ethics Committee
REC reference
17/LO/0387
Date of REC Opinion
12 Apr 2017
REC opinion
Further Information Favourable Opinion