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

Overview

A clinical trial of AAV2/5 vector for patients with Defects in RPE65

Full Title of Study: “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

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Sequential Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: December 2018

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

Interventions

  • Biological: AAV RPE65
    • Comparison of different dosages of AAV RPE65

Arms, Groups and Cohorts

  • Experimental: Low dose AAV-RPE65
    • Subretinal administration of a single low dose of range AAV-RPE65
  • Experimental: Intermediate dose AAV-RPE65
    • Subretinal administration of a single intermediate dose of range AAV-RPE65
  • Experimental: High dose AAV-RPE65
    • Subretinal administration of a single high dose of range AAV-RPE65

Clinical Trial Outcome Measures

Primary Measures

  • Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) – Number of Participants With a Safety Event
    • Time Frame: 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).

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: 3 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • MeiraGTx UK II Ltd
  • Provider of Information About this Clinical Study
    • Sponsor

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