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Completed

NCT Number: NCT02781480

Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)

A clinical trial of AAV2/5 vector for patients with Defects in RPE65

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Key information

Conditions

Leber Congenital Amaurosis Eye Diseases Eye Diseases, Hereditary Retinal Diseases

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

A dose escalation and dose expansion (Phase I/II) trial of adults and children with retinal dystrophy associated with defects in RPE65. ATIMP will be administered to one eye only in a single sub-retinal procedure

Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

  • Aged 3 years or older
  • Early-onset severe retinal dystrophy consistent with RPE65 deficiency

Key Exclusion Criteria:

  • Females who are pregnant or breastfeeding
  • Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.

Treatment and study plan

AAV RPE65

Biological

Comparison of different dosages of AAV RPE65

Primary outcomes

  1. Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event

    Time frame: 6 months

    Safety was defined as an advanced therapy investigational medicinal product (ATIMP) related:

    • Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more.
    • Severe unresponsive inflammation.
    • Infective endophthalmitis.
    • Ocular malignancy.
    • Grade III or above non-ocular suspected unexpected serious adverse reaction (SUSAR).

Sponsors and collaborators

Lead sponsor

MeiraGTx UK II Ltd

Industry

Registry information

Official study title

An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno Associated Virus Vector for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA)

Acronym: OPTIRPE65

Important dates

Study start
2016
Primary completion
2018
Study completion
2018
First posted
May 24, 2016
Registry last updated
Jul 12, 2021

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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