Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3

MeiraGTx UK II Ltd34 enrolled

Overview

This is a longer-term follow-up study of patients with achromatopsia associated with defects in CNGA3 who participated in a clinical trial in which they received AAV-CNGA3 retinal gene therapy, or of patients with achromatopsia associated with defects in CNGB3 who participated in a clinical trial in which they received AAV-CNGB3 retinal gene therapy.

The follow-up study is designed to collect data on the longer-term safety and efficacy of AAV-CNGA3 retinal gene therapy and AAV-CNGB3 retinal gene therapy.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Achromatopsia

Interventions

Prior exposure to AAV-CNGA3 or AAV-CNGB3BIOLOGICAL

Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively.

Eligibility

Sex: ALLMin age: 3 YearsMax age: 100 Years

Medical Language ↔ Plain English

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 the requirements of the study

Locations (2)

Kellogg Eye Center

Ann Arbor, Michigan, United States

Moorfields Eye Hospital NHS Foundation Trust

London, United Kingdom

Outcomes

Primary Outcomes

Incidence of Adverse Events Related to the Treatment

The primary outcome measure is the longer-term safety of treatment with AAV-CNGA3 or AAV-CNGB3, assessed by the absence of IMP-related adverse events.

Time frame: 5 Years

Secondary Outcomes

Improvements in Visual Function as Assessed by Visual Acuity at Month 12

Change from baseline to Month 12 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.

Time frame: 12 months

Improvements in Visual Function as Assessed by Visual Acuity at Month 60

Change from baseline to Month 60 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.

Time frame: 60 months

Improvements in Retinal Function as Assessed by Static Perimetry at Month 12

Change from baseline to Month 12 in contrast sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.

Time frame: 12 months

Improvements in Retinal Function as Assessed by Static Perimetry at Month 60

Change from baseline to Month 60 in contrast sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.

Time frame: 60 months

Quality of Life at Month 12 Measured by QoL Questionnaires in Children and Adolescents

Change from baseline to Month 12 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.

Data from ClinicalTrials.gov

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