Gene Therapy for Achromatopsia (CNGA3)

Achromatopsia Clinical Trial

— CNGA3  

Official title:

An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Children and Adults With Achromatopsia Owing to Defects in CNGA3

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03758404
• Other study ID #: MGT012
• Status: Completed
• Phase: Phase 1/Phase 2
• 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
• 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

Related Conditions & MeSH terms

• Achromatopsia
• Color Vision Defects

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