Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) (OPTIRPE65)

June 17, 2021 updated by: MeiraGTx UK II Ltd

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)

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

Study Overview

Status

Completed

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • London, United Kingdom, EC1V 2PD
        • Moorfields Eye Hospital NHS Foundation Trust
    • Michigan
      • Ann Arbor, Michigan, United States, 48105
        • Kellogg Eye Centre, University of Michigan

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Low dose AAV-RPE65
Subretinal administration of a single low dose of range AAV-RPE65
Comparison of different dosages of AAV RPE65
Experimental: Intermediate dose AAV-RPE65
Subretinal administration of a single intermediate dose of range AAV-RPE65
Comparison of different dosages of AAV RPE65
Experimental: High dose AAV-RPE65
Subretinal administration of a single high dose of range AAV-RPE65
Comparison of different dosages of AAV RPE65

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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).
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2016

Primary Completion (Actual)

December 1, 2018

Study Completion (Actual)

December 1, 2018

Study Registration Dates

First Submitted

April 28, 2016

First Submitted That Met QC Criteria

May 23, 2016

First Posted (Estimate)

May 24, 2016

Study Record Updates

Last Update Posted (Actual)

July 12, 2021

Last Update Submitted That Met QC Criteria

June 17, 2021

Last Verified

June 1, 2021

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Leber Congenital Amaurosis

Clinical Trials on AAV RPE65

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