Gene Therapy for Achromatopsia (CNGB3)

Phase 1

Completed

• Conditions: Achromatopsia
• Interventions: Biological: AAV - CNGB3

• Registration Number: NCT03001310
• Lead Sponsor: MeiraGTx UK II Ltd

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 Timeline

• Study First Submitted: 2016-11-22
• Study First Submitted QC: 2016-12-19
• Study First Posted: 2016-12-23
• Study Start: 2017-01-16
• Primary Completion: 2019-10-25
• Study Completion: 2019-10-25
• Disposition First Submitted: 2020-10-14
• Disposition First Submitted QC: 2020-10-14
• Disposition First Posted: 2020-10-20
• Results First Submitted: 2023-01-03
• Status Verified: 2023-02
• Results First Submitted QC: 2023-02-09
• Last Update Submitted: 2023-02-09
• Results First Posted: 2023-03-08
• Last Update Posted: 2023-03-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 23

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Biological-Low dose AAV - CNGB3	AAV - CNGB3	Subretinal administration of a single low dose of AAV - CNGB3
Biological-High dose AAV - CNGB3	AAV - CNGB3	Subretinal administration of a single high dose of AAV - CNGB3
Biological-Medium dose AAV - CNGB3	AAV - CNGB3	Subretinal administration of a single intermediate dose of AAV - CNGB3

Primary Outcome Measures

Name	Time	Method
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.	6 weeks	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)

Secondary Outcome Measures

Name	Time	Method
Improvements in Visual Function as Assessed by Visual Acuity	6 months	Change from baseline to Week 24 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score in the treated eye. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time.
Improvements in Retinal Function as Assessed by Static Perimetry	6 months	Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.
Quality of Life Measured by QoL Questionnaires in Children and Adolescents	6 months	Change from baseline to Week 24 in EuroQol-5D-Y 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 change from baseline reflects worsening.
Quality of Life Measured by QoL Questionnaires in Adults	6 Months	Change from baseline to Week 24 in EuroQol-5D-5L 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 change from baseline reflects worsening.

Trial Locations

Locations (2)

University of Michigan Kellog Eye Centre; 🇺🇸 Ann Arbor, Michigan, United States

Moorfields Eye Hospital NHS Foundation Trust; 🇬🇧 London, United Kingdom