Gene Therapy for Achromatopsia (CNGB3) (CNGB3)

January 18, 2021 updated by: MeiraGTx UK II Ltd

An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hCARp.hCNGB3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3

A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

CNGB3 retinal gene therapy for patients with achromatopsia

Study Type

Interventional

Enrollment (Actual)

23

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

  • United Kingdom
      • London, United Kingdom
        • Moorfields Eye Hospital NHS Foundation Trust
  • United States
    • Michigan
      • Ann Arbor, Michigan, United States, MI 48105
        • University of Michigan Kellog Eye Centre

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

5 months and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Are aged 3 years or older
  • Have achromatopsia confirmed by a retinal specialist (CI or PI)

Exclusion Criteria:

  • Are females who are pregnant or breastfeeding
  • Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months
  • Have any other condition that the CI/PI considers makes them inappropriate for entry into the trial

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: Biological-Low dose AAV - CNGB3
Subretinal administration of a single low dose of range AAV - CNGB3
Biological: AAV - CNGB3
Comparison of different dosages of AAV-CNGB3
Experimental: Biological-medium dose AAV - CNGB3
Subretinal administration of a single medium dose of range AAV - CNGB3
Biological: AAV - CNGB3
Comparison of different dosages of AAV-CNGB3
Experimental: Biological-high dose AAV - CNGB3
Subretinal administration of a single high dose of range AAV - CNGB3
Biological: AAV - CNGB3
Comparison of different dosages of AAV-CNGB3

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of Adverse Events related to the treatment
Time Frame: 6 months
Safety is defined as the absence of ATIMP-related safety events
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Improvement in visual function
Time Frame: 6 months
Improvements in visual function as assessed by visual assessment
6 months
Improvement in retinal function
Time Frame: 6 months
Improvements in retinal function as assessed by visual assessment
6 months
Improvement in Quality of life
Time Frame: 6 months
Quality of life will be measured by the QoL questionnaire
6 months

Collaborators and Investigators

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

Sponsor

MeiraGTx UK II Ltd

Collaborators

Syne Qua Non Limited
EMAS Pharma

Investigators

  • Principal Investigator: James Bainbridge, Prof, Chief Investigator

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 (Actual)

January 16, 2017

Primary Completion (Actual)

October 25, 2019

Study Completion (Actual)

October 25, 2019

Study Registration Dates

First Submitted

November 22, 2016

First Submitted That Met QC Criteria

December 19, 2016

First Posted (Estimate)

December 23, 2016

Study Record Updates

Last Update Posted (Actual)

January 22, 2021

Last Update Submitted That Met QC Criteria

January 18, 2021

Last Verified

January 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MGT006
  • 2016-002290-35 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

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.

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