Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
OPTIRPE65
An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno Associated Virus Vector for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA)
1 other identifier
interventional
15
2 countries
2
Brief Summary
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Apr 2016
Typical duration for phase_1
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 1, 2016
CompletedFirst Submitted
Initial submission to the registry
April 28, 2016
CompletedFirst Posted
Study publicly available on registry
May 24, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2018
CompletedResults Posted
Study results publicly available
April 8, 2021
CompletedJuly 12, 2021
June 1, 2021
2.7 years
April 28, 2016
March 10, 2021
June 17, 2021
Conditions
Outcome Measures
Primary Outcomes (1)
Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event
Safety was defined as an advanced therapy investigational medicinal product (ATIMP) related: * Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more. * Severe unresponsive inflammation. * Infective endophthalmitis. * Ocular malignancy. * Grade III or above non-ocular suspected unexpected serious adverse reaction (SUSAR).
6 months
Study Arms (3)
Low dose AAV-RPE65
EXPERIMENTALSubretinal administration of a single low dose of range AAV-RPE65
Intermediate dose AAV-RPE65
EXPERIMENTALSubretinal administration of a single intermediate dose of range AAV-RPE65
High dose AAV-RPE65
EXPERIMENTALSubretinal administration of a single high dose of range AAV-RPE65
Interventions
Comparison of different dosages of AAV RPE65
Eligibility Criteria
You may qualify if:
- Aged 3 years or older
- Early-onset severe retinal dystrophy consistent with RPE65 deficiency
You may not qualify if:
- Females who are pregnant or breastfeeding
- Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (2)
Kellogg Eye Centre, University of Michigan
Ann Arbor, Michigan, 48105, United States
Moorfields Eye Hospital NHS Foundation Trust
London, EC1V 2PD, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Chief Medical Officer
- Organization
- MeiraGTx
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 28, 2016
First Posted
May 24, 2016
Study Start
April 1, 2016
Primary Completion
December 1, 2018
Study Completion
December 1, 2018
Last Updated
July 12, 2021
Results First Posted
April 8, 2021
Record last verified: 2021-06