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Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and 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. 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 : March 24, 2021
Sponsor:
Collaborators:
Syne Qua Non Limited
EMAS Pharma
Information provided by (Responsible Party):
MeiraGTx UK II Ltd

Tracking Information
First Submitted Date  ICMJE August 16, 2017
First Posted Date  ICMJE September 12, 2017
Last Update Posted Date March 24, 2021
Actual Study Start Date  ICMJE June 27, 2017
Estimated Primary Completion Date July 10, 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 7, 2017)
Incidence of Adverse Events related to the treatment [ Time Frame: 5 Years ]
longer term safety as the absence of ATIMP-related safety events
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 7, 2017)
  • Improvement in the visual function [ Time Frame: 5 years ]
    Improvements in visual function as assessed by visual assessment
  • Improvement in retinal function [ Time Frame: 5 years ]
    Improvement in the retinal function as assessed by visual assessment
  • Improvement in quality of life [ Time Frame: 5 years ]
    Quality of life will be measured by QoL questionnaires
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3
Official Title  ICMJE 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 (AAV2/8-hCARp.hCNGB3 and AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3 or CNGA3
Brief Summary This study is a longer-term follow-up study for patients who participated in one of the clinical trials: AAV - CNGB3 retinal gene therapy for patients with achromatopsia, or AAV - CNGA3 retinal gene therapy for patients with achromatopsia.
Detailed Description The follow up study is designed to collect data on longer-term safety and efficacy.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Intervention Model Description:
Follow-up of patients from two separate trials but there is no difference in follow-up assessments for the patients therefore they can be considered a "single group"
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Achromatopsia
Intervention  ICMJE Biological: either AAV - CNGB3 or AAV - CNGA3
Comparison of different dosages of either AAV - CNGB3 or AAV - CNGA3
Study Arms  ICMJE
  • Experimental: Biological-Low dose of either AAV - CNGB3 or AAV - CNGA3
    Subretinal administration of a single low dose of either AAV - CNGB3 or AAV - CNGA3
    Intervention: Biological: either AAV - CNGB3 or AAV - CNGA3
  • Experimental: Biological-medium dose of either AAV - CNGB3 or AAV - CNGA3
    Subretinal administration of a single medium dose of either AAV - CNGB3 or AAV - CNGA3
    Intervention: Biological: either AAV - CNGB3 or AAV - CNGA3
  • Experimental: Biological-high dose of either AAV - CNGB3 or AAV - CNGA3
    Subretinal administration of a single high dose of either AAV - CNGB3 or AAV - CNGA3
    Intervention: Biological: either AAV - CNGB3 or 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 Recruiting
Estimated Enrollment  ICMJE
 (submitted: September 10, 2019)
72
Original Estimated Enrollment  ICMJE
 (submitted: September 7, 2017)
18
Estimated Study Completion Date  ICMJE August 7, 2024
Estimated Primary Completion Date July 10, 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion in the study will be limited to individuals who:

  1. Are able to give informed consent or assent, with or without the guidance of their parent(s)/guardian(s) where appropriate
  2. Received AAV2/8-hCARp.hCNGB3 or AAV2/8-hG1.7p.coCNGA3 by intraocular administration in the prior open-label, Phase I/II, dose escalation study (EudraCT 2016-002290-35 or EudraCT 2018-003431-29)
  3. Are willing to adhere to the protocol and long-term follow-up

Individuals will be excluded who:

Are unwilling or unable to meet with the requirements of the study

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years to 100 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: MeiraGTx UK II Ltd +44 (0)20 3866 4320 ocularinfo@meiragtx.com
Listed Location Countries  ICMJE United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03278873
Other Study ID Numbers  ICMJE MGT007
2016-003856-59 ( EudraCT Number )
Has Data Monitoring Committee No
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
  • Syne Qua Non Limited
  • EMAS Pharma
Investigators  ICMJE Not Provided
PRS Account MeiraGTx UK II Ltd
Verification Date March 2021

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