Clinical Trials

Date: 2017-02-08

Type of information: update on patient enrollment

phase: 1-2

Announcement: update

Company: MeiraGTx (USA - NY)

Product: A001 - adenovirus associated viral vector serotype 5 containing the human RPE65 gene

Action mechanism: gene therapy.

Disease: Leber’s Congenital Amaurosis with RPE65 mutations

Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases

Country: UK

Trial details:

This clinical trial of gene therapy for the treatment of Leber Congenital Amaurosis (LCA) (OPTIRPE65) is a clinical trial of AAV2/5 vector for patients with defects in RPE65.
The primary endpoint of the study is to determine the safety of the treatment. Secondary endpoints include improvement in visual function including a mobility maze, retinal function, retinal structure and quality of life measures.
(NCT02781480)

Latest news:

• On December 7, 2017, MeiraGTx announced that the company has completed the three dose escalation cohorts of adult patients in this study and in November 2017 the first pediatric patient was treated at the Moorfields Eye Hospital in London.
In October 2017, the FDA accepted the Investigational New Drug (IND) application for A001 opening the path for the treatment of pediatric patients in the U.S.
• On February 8, 2017, MeiraGTx announced that the company has successfully completed dosing of patients in the second cohort in its clinical trial for Leber’s Congenital Amaurosis with RPE65 mutations at the Moorfields Eye Hospital in London.