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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
Results First Posted : February 11, 2022
Last Update Posted : February 11, 2022
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
Results First Submitted Date  ICMJE January 6, 2022
Results First Posted Date  ICMJE February 11, 2022
Last Update Posted Date February 11, 2022
Actual Study Start Date  ICMJE August 12, 2019
Actual Primary Completion Date June 10, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 10, 2022)
Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Products (ATIMP), Not Surgery Alone. [ Time Frame: 6 Weeks ]
The primary outcome is defined as any of the below occurring during the 6 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Products (ATIMP), not surgery alone:
  • Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more that fails to resolve to within 15 letters of baseline in a 4-week period once prophylactic treatment commences
  • Severe unresponsive inflammation
  • Infective endophthalmitis
  • Ocular malignancy
  • Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)
Original 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
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 6, 2022)
  • 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 adeno-associated virus vector (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: adeno-associated virus vector AAV- CNGA3
Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene
Study Arms  ICMJE
  • Experimental: Low dose adeno-associated virus (AAV) CNGA3
    Subretinal administration of a single low dose AAV CNGA3
    Intervention: Biological: adeno-associated virus vector AAV- CNGA3
  • Experimental: Intermediate dose adeno-associated virus (AAV) CNGA3
    Subretinal administration of a single intermediate dose AAV CNGA3
    Intervention: Biological: adeno-associated virus vector AAV- CNGA3
  • Experimental: High dose adeno-associated virus (AAV) CNGA3
    Subretinal administration of a single high dose AAV CNGA3
    Intervention: Biological: adeno-associated virus vector 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 investigator

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 investigator 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
Current Responsible Party MeiraGTx UK II Ltd
Original Responsible Party Same as current
Current Study Sponsor  ICMJE MeiraGTx UK II Ltd
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Janssen Research & Development, LLC
Investigators  ICMJE
Principal Investigator: James Bainbridge Chief Investigator
PRS Account MeiraGTx UK II Ltd
Verification Date February 2022

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