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Gene Therapy for Achromatopsia (CNGA3) (CNGA3)

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03758404
Recruitment Status : Completed
First Posted : November 29, 2018
Last Update Posted : June 25, 2021
Sponsor:
Collaborator:
Janssen Research & Development, LLC
Information provided by (Responsible Party):
MeiraGTx UK II Ltd

Tracking Information
First Submitted Date  ICMJE November 27, 2018
First Posted Date  ICMJE November 29, 2018
Last Update Posted Date June 25, 2021
Actual Study Start Date  ICMJE July 18, 2019
Actual Primary Completion Date June 10, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 28, 2018)
Incidence of Adverse Events related to the treatment [ Time Frame: 6 Months ]
Safety is defined as the absence of ATIMP-related safety events
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 25, 2019)
  • Improvement in visual function [ Time Frame: 6 Months ]
    Improvements in visual function as assessed by visual acuity
  • Improvement in retinal function [ Time Frame: 6 Months ]
    Improvements in retinal function as assessed by microperimetry and perimetry
  • Improvement in Quality of life [ Time Frame: 6 Months ]
    Quality of life will be measured by QoL questionnaires
Original Secondary Outcome Measures  ICMJE
 (submitted: November 28, 2018)
  • Improvement in visual function [ Time Frame: 6 Months ]
    Improvements in visual function as assessed by visual acuity
  • Improvement in retinal function [ Time Frame: 6 Months ]
    Improvements in retinal function as assessed by microperimetry and perimetry
  • Improvement in Quality of life [ Time Frame: 6 Months ]
    Quality of life will be measured by the QoL questionnaire
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Gene Therapy for Achromatopsia (CNGA3)
Official Title  ICMJE An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Children and Adults With Achromatopsia Owing to Defects in CNGA3
Brief Summary A clinical trial of AAV - CNGA3 retinal gene therapy for patients with achromatopsia
Detailed Description CNGA3 retinal gene therapy for patients with achromatopsia
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Achromatopsia
Intervention  ICMJE Biological: AAV- CNGA3
AAV gene therapy for defects in CNGA3 gene
Study Arms  ICMJE
  • Experimental: Low dose AAV - CNGA3
    Subretinal administration of a single low dose of range AAV - CNGA3
    Intervention: Biological: AAV- CNGA3
  • Experimental: Intermediate dose AAV - CNGA3
    Subretinal administration of a single intermediate dose of range AAV - CNGA3
    Intervention: Biological: AAV- CNGA3
  • Experimental: High dose AAV - CNGA3
    Subretinal administration of a single high dose of range AAV - CNGA3
    Intervention: Biological: AAV- CNGA3
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 24, 2021)
11
Original Estimated Enrollment  ICMJE
 (submitted: November 28, 2018)
18
Actual Study Completion Date  ICMJE June 10, 2021
Actual Primary Completion Date June 10, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Are aged years or over
  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03758404
Other Study ID Numbers  ICMJE MGT012
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party MeiraGTx UK II Ltd
Study Sponsor  ICMJE MeiraGTx UK II Ltd
Collaborators  ICMJE Janssen Research & Development, LLC
Investigators  ICMJE
Principal Investigator: James Bainbridge Chief Investigator
PRS Account MeiraGTx UK II Ltd
Verification Date June 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP