Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)

Phase 1

Completed

• Conditions: Leber Congenital Amaurosis
• Interventions: Biological: AAV RPE65

• Registration Number: NCT02781480
• Lead Sponsor: MeiraGTx UK II Ltd

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

Study Timeline

• Study Start: 2016-04
• Study First Submitted: 2016-04-28
• Study First Submitted QC: 2016-05-23
• Study First Posted: 2016-05-24
• Primary Completion: 2018-12
• Study Completion: 2018-12
• Results First Submitted: 2021-03-10
• Results First Submitted QC: 2021-03-10
• Results First Posted: 2021-04-08
• Status Verified: 2021-06
• Last Update Submitted: 2021-06-17
• Last Update Posted: 2021-07-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Low dose AAV-RPE65	AAV RPE65	Subretinal administration of a single low dose of range AAV-RPE65
High dose AAV-RPE65	AAV RPE65	Subretinal administration of a single high dose of range AAV-RPE65
Intermediate dose AAV-RPE65	AAV RPE65	Subretinal administration of a single intermediate dose of range AAV-RPE65

Primary Outcome Measures

Name	Time	Method
Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event	6 months	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).

Trial Locations

Locations (2)

Kellogg Eye Centre, University of Michigan; 🇺🇸 Ann Arbor, Michigan, United States

Moorfields Eye Hospital NHS Foundation Trust; 🇬🇧 London, United Kingdom