NCT00643747

Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: University College, London · Academic / Other
• Sex: All
• Age: 5 Years – 30 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Retinal Degeneration

Interventions

Type	Name	Description
BIOLOGICAL	tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)	Single subretinal injection of vector suspension; up to 3x10e12 vector particles

Timeline

• Start date: 2007-01-01
• Primary completion: 2014-12-01
• Completion: 2014-12-01
• First posted: 2008-03-26
• Last updated: 2015-12-07

Locations

1 site across 1 country: United Kingdom