ClinicalTrials.gov
ClinicalTrials.gov Menu

Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03278873
Recruitment Status : Recruiting
First Posted : September 12, 2017
Last Update Posted : December 18, 2017
Sponsor:
Collaborators:
Syne Qua Non Limited
EMAS Pharma
Information provided by (Responsible Party):
MeiraGTx UK II Ltd

Brief Summary:
This study is a longer-term follow-up study for patients who participated in a clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia.

Condition or disease Intervention/treatment Phase
Achromatopsia Biological: AAV - CNGB3 Phase 1 Phase 2

Detailed Description:
The follow up study is designed to collect data on longer-term safety and efficacy.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3
Actual Study Start Date : June 27, 2017
Estimated Primary Completion Date : July 10, 2023
Estimated Study Completion Date : August 7, 2023

Resource links provided by the National Library of Medicine

U.S. FDA Resources

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



Primary Outcome Measures :
  1. Incidence of Adverse Events related to the treatment [ Time Frame: 5 Years ]
    longer term safety as the absence of ATIMP-related safety events


Secondary Outcome Measures :
  1. Improvement in the visual function [ Time Frame: 5 years ]
    Improvements in visual function as assessed by visual assessment

  2. Improvement in retinal function [ Time Frame: 5 years ]
    Improvement in the retinal function as assessed by visual assessment

  3. Improvement in quality of life [ Time Frame: 5 years ]
    Quality of life will be measured by QoL questionnaires



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   3 Years to 100 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Were enrolled and treated in the CNGB3 gene therapy trial for achromatopsia
  • Are willing to adhere to the protocol and long-term follow-up

Exclusion Criteria:

  • Individuals will be excluded if they are unwilling or unable to meet with the requirements of the study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03278873


Contacts
Contact: Julie Bakobaki +44 (0)20 3866 4320 julie.bakobaki@meiragtx.com
Contact: Anna Morka anna.morka@meiragtx.com

Locations
United Kingdom
Moorfields Eye Hospital NHS Foundation Trust Recruiting
London, United Kingdom
Contact: Michel Michaelides       michel.michaelides@ucl.ac.uk   
Principal Investigator: James Bainbridge, Prof         
Sub-Investigator: Michel Michaelides, Prof         
Sponsors and Collaborators
MeiraGTx UK II Ltd
Syne Qua Non Limited
EMAS Pharma

Responsible Party: MeiraGTx UK II Ltd
ClinicalTrials.gov Identifier: NCT03278873     History of Changes
Other Study ID Numbers: MGT007
2016-003856-59 ( EudraCT Number )
First Posted: September 12, 2017    Key Record Dates
Last Update Posted: December 18, 2017
Last Verified: December 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Color Vision Defects
Vision Disorders
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases
Eye Diseases
Signs and Symptoms