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Completed

NCT Number: NCT00643747

Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

The purpose of the study is to determine whether gene therapy is safe and effective for the treatment of severe childhood blindness caused by mutations in RPE65.

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Key information

Conditions

Retinal Degeneration Eye Diseases Eye Diseases, Hereditary Leber Congenital Amaurosis Retinal Diseases Retinal Dystrophies

Age range

5 year–30 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Moorfields Eye Hospital NHS Foundation Trust

London, EC1V 2PD, United Kingdom

About this study

The main objective of the proposed trial is to determine the safety and efficacy subretinal administration of a recombinant adeno-associated viral vector (rAAV 2/2.hRPE65p.hRPE65) at three different dosage levels in individuals with autosomal recessive severe early-onset retinal degeneration due to mutations in RPE65. We have a comprehensive clinical monitoring plan to investigate the safety and efficacy of vector delivery.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Clinical diagnosis of severe early-onset retinal dystrophy confirmed missense mutation(s) in RPE65

Exclusion criteria

  • Visual acuity in the study eye better than 6/36 Snellen
  • Hypertension
  • Diabetes mellitus
  • Tuberculosis
  • Renal impairment
  • Immunocompromise
  • Osteoporosis
  • Gastric ulceration
  • Severe affective disorder)
  • Pregnancy or lactation

Treatment and study plan

tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)

Biological

Single subretinal injection of vector suspension; up to 3x10e12 vector particles

Other names: rAAV 2/2.hRPE65p.hRPE65

Primary outcomes

  1. intraocular inflammation

    Time frame: at intervals up to 12 months

Secondary outcomes

  1. visual function

    Time frame: intervals up to 12 months

Sponsors and collaborators

Lead sponsor

University College, London

Other

Collaborators

  • Moorfields Eye Hospital NHS Foundation Trust
  • Targeted Genetics Corporation

Registry information

Official study title

An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (AAV2/2-hRPE65p-hRPE65) for Gene Therapy of Severe Early-onset Retinal Degeneration

Important dates

Study start
2007
Primary completion
2014
Study completion
2014
First posted
Mar 26, 2008
Registry last updated
Dec 7, 2015

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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