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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03278873
Other study ID # MGT007
Secondary ID 2016-003856-59
Status Completed
Phase Phase 1/Phase 2
First received
Last updated
Start date June 29, 2017
Est. completion date April 4, 2024

Study information

Verified date May 2024
Source MeiraGTx UK II Ltd
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Description:

The follow up study is designed to collect data on longer-term safety and efficacy.


Recruitment information / eligibility

Status Completed
Enrollment 34
Est. completion date April 4, 2024
Est. primary completion date April 4, 2024
Accepts healthy volunteers No
Gender All
Age group 3 Years to 100 Years
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
either AAV - CNGB3 or AAV - CNGA3
Comparison of different dosages of either AAV - CNGB3 or AAV - CNGA3

Locations

Country Name City State
United Kingdom Moorfields Eye Hospital NHS Foundation Trust London
United States Kellog Eye Center Ann Arbor Michigan

Sponsors (3)

Lead Sponsor Collaborator
MeiraGTx UK II Ltd EMAS Pharma, Syne Qua Non Limited

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Incidence of Adverse Events related to the treatment longer term safety as the absence of ATIMP-related safety events 5 Years
Secondary Improvement in the visual function Improvements in visual function as assessed by visual assessment 5 years
Secondary Improvement in retinal function Improvement in the retinal function as assessed by visual assessment 5 years
Secondary Improvement in quality of life Quality of life will be measured by QoL questionnaires 5 years
See also
  Status Clinical Trial Phase
Completed NCT04124185 - Natural History Study for Achromatopsia
Active, not recruiting NCT02610582 - Safety and Efficacy of rAAV.hCNGA3 Gene Therapy in Patients With CNGA3-linked Achromatopsia Phase 1/Phase 2
Not yet recruiting NCT04041232 - PBA Use for Treatment of ATF6-/- Patients Early Phase 1
Recruiting NCT02435940 - Inherited Retinal Degenerative Disease Registry
Completed NCT01846052 - Clinical and Genetic Characterization of Individuals With Achromatopsia N/A
Active, not recruiting NCT01648452 - CNTF Implants for CNGB3 Achromatopsia Phase 1/Phase 2
Completed NCT03001310 - Gene Therapy for Achromatopsia (CNGB3) Phase 1/Phase 2
Active, not recruiting NCT02935517 - Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial) Phase 1/Phase 2
Completed NCT03758404 - Gene Therapy for Achromatopsia (CNGA3) Phase 1/Phase 2
Active, not recruiting NCT02599922 - Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial) Phase 1/Phase 2