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Completed

NCT Number: NCT03001310

Gene Therapy for Achromatopsia (CNGB3)

A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia

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Key information

Conditions

Achromatopsia Color Vision Defects Cone Dystrophy Eye Diseases Eye Diseases, Hereditary Nervous System Diseases Neurologic Manifestations Pathological Conditions, Signs and Symptoms Sensation Disorders Signs and Symptoms Vision Disorders

Age range

3 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Moorfields Eye Hospital NHS Foundation Trust, London, United Kingdom

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About this study

CNGB3 retinal gene therapy for patients with achromatopsia

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

AAV - CNGB3

Biological

Comparison of different dosages of AAV-CNGB3

Primary outcomes

  1. 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.

    Time frame: 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 outcomes

  1. Improvements in Visual Function as Assessed by Visual Acuity

    Time frame: 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.

  2. Improvements in Retinal Function as Assessed by Static Perimetry

    Time frame: 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.

  3. Quality of Life Measured by QoL Questionnaires in Children and Adolescents

    Time frame: 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.

  4. Quality of Life Measured by QoL Questionnaires in Adults

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

MeiraGTx UK II Ltd

Industry

Collaborators

  • Janssen Research & Development, LLC

Registry information

Official study title

An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hCARp.hCNGB3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3

Acronym: CNGB3

Important dates

Study start
2017
Primary completion
2019
Study completion
2019
First posted
Dec 23, 2016
Registry last updated
Mar 8, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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