Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) (OPTIRPE65)
Primary Purpose
Leber Congenital Amaurosis
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
AAV RPE65
Sponsored by
About this trial
This is an interventional treatment trial for Leber Congenital Amaurosis
Eligibility Criteria
Key Inclusion Criteria:
- Aged 3 years or older
- Early-onset severe retinal dystrophy consistent with RPE65 deficiency
Key Exclusion Criteria:
- Females who are pregnant or breastfeeding
- Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.
Sites / Locations
- Kellogg Eye Centre, University of Michigan
- Moorfields Eye Hospital NHS Foundation Trust
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
Low dose AAV-RPE65
Intermediate dose AAV-RPE65
High dose AAV-RPE65
Arm Description
Subretinal administration of a single low dose of range AAV-RPE65
Subretinal administration of a single intermediate dose of range AAV-RPE65
Subretinal administration of a single high dose of range AAV-RPE65
Outcomes
Primary Outcome Measures
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).
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02781480
Brief Title
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
Acronym
OPTIRPE65
Official Title
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)
Study Type
Interventional
2. Study Status
Record Verification Date
June 2021
Overall Recruitment Status
Completed
Study Start Date
April 2016 (undefined)
Primary Completion Date
December 2018 (Actual)
Study Completion Date
December 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
MeiraGTx UK II Ltd
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
Detailed Description
A dose escalation and dose expansion (Phase I/II) trial of adults and children with retinal dystrophy associated with defects in RPE65. ATIMP will be administered to one eye only in a single sub-retinal procedure
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leber Congenital Amaurosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Model Description
AAV2/5-OPTIRPE65
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
15 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Low dose AAV-RPE65
Arm Type
Experimental
Arm Description
Subretinal administration of a single low dose of range AAV-RPE65
Arm Title
Intermediate dose AAV-RPE65
Arm Type
Experimental
Arm Description
Subretinal administration of a single intermediate dose of range AAV-RPE65
Arm Title
High dose AAV-RPE65
Arm Type
Experimental
Arm Description
Subretinal administration of a single high dose of range AAV-RPE65
Intervention Type
Biological
Intervention Name(s)
AAV RPE65
Intervention Description
Comparison of different dosages of AAV RPE65
Primary Outcome Measure Information:
Title
Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event
Description
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).
Time Frame
6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
Aged 3 years or older
Early-onset severe retinal dystrophy consistent with RPE65 deficiency
Key Exclusion Criteria:
Females who are pregnant or breastfeeding
Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.
Facility Information:
Facility Name
Kellogg Eye Centre, University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48105
Country
United States
Facility Name
Moorfields Eye Hospital NHS Foundation Trust
City
London
ZIP/Postal Code
EC1V 2PD
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
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