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

Plain-language summary

This study evaluated the safety and effect of a gene therapy called AAV RPE65 in patients with Leber congenital amaurosis (LCA) caused by RPE65 deficiency. It included people aged 3 and older with early-onset severe retinal dystrophy consistent with RPE65 deficiency. It was conducted in Phase 1/Phase 2 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Completed

Sponsor

MeiraGTx UK II Ltd

Intervention

AAV RPE65

Start date

4/1/2016

Estimated completion

12/1/2018

Countries

United States, United Kingdom

Related genes

Locations

• Ann Arbor, United States
• London, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.