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Gene Therapy for Achromatopsia (CNGB3)

Sponsor
MeiraGTx UK II Ltd (Industry)
Overall Status
Completed
CT.gov ID
NCT03001310
Collaborator
EMAS Pharma (Industry), Syne Qua Non Limited (Industry)
23
Enrollment
2
Locations
3
Arms
33.2
Actual Duration (Months)
11.5
Patients Per Site
0.3
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia

Condition or Disease Intervention/Treatment Phase
  • Biological: AAV - CNGB3
 Phase 1/Phase 2

Detailed Description

CNGB3 retinal gene therapy for patients with achromatopsia

Study Design

Study Type:
Interventional
Actual Enrollment :
23 participants
Allocation:
Non-Randomized
Intervention Model:
Sequential 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
Actual Study Start Date :
Jan 16, 2017
Actual Primary Completion Date :
Oct 25, 2019
Actual Study Completion Date :
Oct 25, 2019

Arms and Interventions

Arm Intervention/Treatment
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

Outcome Measures

Primary Outcome Measures

  1. Incidence of Adverse Events related to the treatment [6 months]

    Safety is defined as the absence of ATIMP-related safety events

Secondary Outcome Measures

  1. Improvement in visual function [6 months]

    Improvements in visual function as assessed by visual assessment

  2. Improvement in retinal function [6 months]

    Improvements in retinal function as assessed by visual assessment

  3. Improvement in Quality of life [6 months]

    Quality of life will be measured by the QoL questionnaire

Eligibility Criteria

Criteria

Ages Eligible for Study:
3 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Locations

Site City State Country Postal Code
1 University of Michigan Kellog Eye Centre Ann Arbor Michigan United States MI 48105
2 Moorfields Eye Hospital NHS Foundation Trust London United Kingdom

Sponsors and Collaborators

  • MeiraGTx UK II Ltd
  • EMAS Pharma
  • Syne Qua Non Limited

Investigators

  • Principal Investigator: James Bainbridge, Prof, Chief Investigator

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
MeiraGTx UK II Ltd
ClinicalTrials.gov Identifier:
NCT03001310
Other Study ID Numbers:
  • MGT006
  • 2016-002290-35
First Posted:
Dec 23, 2016
Last Update Posted:
Jan 22, 2021
Last Verified:
Jan 1, 2021
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Color Vision Defects

Study Results

No Results Posted as of Jan 22, 2021