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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03758404
Other study ID # MGT012
Secondary ID
Status Completed
Phase Phase 1/Phase 2
First received
Last updated
Start date August 12, 2019
Est. completion date June 10, 2021

Study information

Verified date November 2022
Source MeiraGTx UK II Ltd
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia


Description:

CNGA3 retinal gene therapy for patients with achromatopsia


Recruitment information / eligibility

Status Completed
Enrollment 11
Est. completion date June 10, 2021
Est. primary completion date June 10, 2021
Accepts healthy volunteers No
Gender All
Age group 3 Years and older
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
adeno-associated virus vector AAV- CNGA3
Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene

Locations

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

Sponsors (2)

Lead Sponsor Collaborator
MeiraGTx UK II Ltd Janssen Research & Development, LLC

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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. 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)
6 Weeks
Secondary Improvements in Visual Function as Assessed by Visual Acuity Change from baseline to Week 24 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time. 6 Months
Secondary Improvements in Retinal Function as Assessed by Static Perimetry Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity. 6 Months
Secondary Quality of Life Measured by QoL Questionnaires in Children and Adolescents Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in children and adolescents. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening. 6 Months
Secondary Quality of Life Measured by QoL Questionnaires in Adults Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in adults. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening. 6 Months
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
Completed NCT03278873 - Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3 Phase 1/Phase 2
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
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