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Gene Therapy for Achromatopsia (CNGB3) (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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03001310
Recruitment Status : Active, not recruiting
First Posted : December 23, 2016
Last Update Posted : September 13, 2019
Sponsor:
Collaborators:
EMAS Pharma
Syne Qua Non Limited
Information provided by (Responsible Party):
MeiraGTx UK II Ltd

Tracking Information
First Submitted Date  ICMJE November 22, 2016
First Posted Date  ICMJE December 23, 2016
Last Update Posted Date September 13, 2019
Actual Study Start Date  ICMJE January 12, 2016
Actual Primary Completion Date May 8, 2019   (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: 6 months ]
Safety is defined as the absence of ATIMP-related safety events
Original Primary Outcome Measures  ICMJE
 (submitted: December 19, 2016)
Incidence of Adverse Events related to the sub retinal administration of an AAV2/8 vector. [ Time Frame: 6 months ]
Safety is defined as the absence of ATIMP-related
  • Reduction in visual acuity by 15 ETDRS letters or more
  • Severe unresponsive inflammation
  • Infective endophthalmitis
  • Ocular malignancy
  • Grade III or above non-ocular SUSAR
Change History Complete list of historical versions of study NCT03001310 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 7, 2017)
  • 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
Original Secondary Outcome Measures  ICMJE
 (submitted: December 19, 2016)
  • Improvement in the visual function [ Time Frame: 6 months ]
    • Any improvements in visual function from baseline that are greater than the test-retest variation and are sustained for at least two consecutive assessments.
  • Improvement in retinal function [ Time Frame: 6 months ]
    • Any improvement in retinal function from baseline that is measurable by electroretinography (ERG).
  • Improvement in Quality of life [ Time Frame: 6 months ]
    Quality of life is measured by the Impact of Visual Impairment (IVI) questionnaire and the EQ5D-5L
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 (CNGB3)
Official Title  ICMJE 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
Brief Summary A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia
Detailed Description CNGB3 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 - CNGB3
Comparison of different dosages of AAV-CNGB3
Study Arms  ICMJE
  • Experimental: Biological-Low dose AAV - CNGB3
    Subretinal administration of a single low dose of range AAV - CNGB3
    Intervention: Biological: AAV - CNGB3
  • Experimental: Biological-medium dose AAV - CNGB3
    Subretinal administration of a single medium dose of range AAV - CNGB3
    Intervention: Biological: AAV - CNGB3
  • Experimental: Biological-high dose AAV - CNGB3
    Subretinal administration of a single high dose of range AAV - CNGB3
    Intervention: Biological: AAV - CNGB3
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 Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: September 12, 2019)
23
Original Estimated Enrollment  ICMJE
 (submitted: December 19, 2016)
18
Estimated Study Completion Date  ICMJE December 8, 2019
Actual Primary Completion Date May 8, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
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
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03001310
Other Study ID Numbers  ICMJE MGT006
2016-002290-35 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party MeiraGTx UK II Ltd
Study Sponsor  ICMJE MeiraGTx UK II Ltd
Collaborators  ICMJE
  • EMAS Pharma
  • Syne Qua Non Limited
Investigators  ICMJE
Principal Investigator: James Bainbridge, Prof Chief Investigator
PRS Account MeiraGTx UK II Ltd
Verification Date September 2019

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