SOLSTICE
Research type
Research Study
Full title
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Treated Previously with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) in an Antecedent Study
IRAS ID
239505
Contact name
Robert MacLaren
Contact email
Sponsor organisation
NightstaRx Ltd
Eudract number
2017-003104-42
Duration of Study in the UK
7 years, 7 months, 0 days
Research summary
This is a prospective and part restrospective multi-centre, observational, follow-up study for subjects who have previously received a sub-retinal injection of AAV2-REP1 for the treatment of CHM in an antecedent study. No further gene therapy will be given in this protocol. Subjects will rollover into the current study when they complete the antecedent study. Visit 1 of the current study should wherever possible be combined with the last visit of the antecedent study.
The current study will consist of up to 9 visits over a maximum 48-month study period. Visit 1 will be the final visit in the antecedent study. All subsequent visits will be determined according to the date of surgery in the antecedent study: Visit 2 (1.5 years post-surgery); Visit 3 (2.0 years post-surgery); Visit 4 (2.5 years post-surgery); Visit 5 (3.0 years post-surgery); Visit 6 (3.5 years post-surgery); Visit 7 (4.0 years post-surgery); Visit 8 (4.5 years post-surgery) and Visit 9 (5.0 years post-surgery). If subjects are followed for > 1 year post-treatment in their antecedent study, they will not complete the corresponding visit(s) in the current study and will move to the next availa-ble post-treatment follow-up timepoint.
If a subject withdraws early from the study, every reasonable effort should be made to complete the assessments scheduled for the Early Termination Visit.REC name
South Central - Oxford A Research Ethics Committee
REC reference
18/SC/0206
Date of REC Opinion
26 Jun 2018
REC opinion
Further Information Favourable Opinion