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Gene Therapy for Achromatopsia (CNGB3) (CNGB3)

This study is currently recruiting participants.
Verified September 2017 by MeiraGTx UK II Ltd
Sponsor:
ClinicalTrials.gov Identifier:
NCT03001310
First Posted: December 23, 2016
Last Update Posted: September 11, 2017
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.
Collaborators:
Emas Pharma Ltd.
Syne Qua Non Limited
Information provided by (Responsible Party):
MeiraGTx UK II Ltd
  Purpose
A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia

Condition Intervention Phase
Achromatopsia Biological: AAV - CNGB3 Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by MeiraGTx UK II Ltd:

Primary Outcome Measures:
  • Incidence of Adverse Events related to the treatment [ Time Frame: 6 months ]
    Safety is defined as the absence of ATIMP-related safety events


Secondary Outcome Measures:
  • Improvement in visual function [ Time Frame: 6 months ]
    Improvements in visual function as assessed by visual assessment

  • Improvement in retinal function [ Time Frame: 6 months ]
    Improvements in retinal function as assessed by visual assessment

  • Improvement in Quality of life [ Time Frame: 6 months ]
    Quality of life will be measured by the QoL questionnaire


Estimated Enrollment: 18
Actual Study Start Date: January 12, 2016
Estimated Study Completion Date: February 2019
Estimated Primary Completion Date: February 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

Detailed Description:
CNGB3 retinal gene therapy for patients with achromatopsia
  Eligibility

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 and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
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): NCT03001310


Contacts
Contact: MeiraGTx UK II Ltd +44 (0)20 3866 4320 ocularinfo@meiragtx.com

Locations
United Kingdom
Moorfields Eye Hospital NHS Foundation Trust Recruiting
London, United Kingdom
Contact: Sophie Connor    02075662821    Sophie.Connor@moorfields.nhs.uk   
Contact: Nashila Hirji, Dr       Nashila.Hirji@moorfields.nhs.uk   
Principal Investigator: James Bainbridge, Prof         
Sponsors and Collaborators
MeiraGTx UK II Ltd
Emas Pharma Ltd.
Syne Qua Non Limited
Investigators
Principal Investigator: James Bainbridge, Prof Chief Investigator
  More Information

Responsible Party: MeiraGTx UK II Ltd
ClinicalTrials.gov Identifier: NCT03001310     History of Changes
Other Study ID Numbers: MGT006
2016-002290-35 ( EudraCT Number )
First Submitted: November 22, 2016
First Posted: December 23, 2016
Last Update Posted: September 11, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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