Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations

LCA (Leber Congenital Amaurosis) Clinical Trial

Official title: Phase 1b Study to Evaluate QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT)

Status Completed

Clinical Trial Summary

The purpose of this study is:

• to evaluate the safety of oral QLT091001

• to evaluate whether 7-day treatment with oral QLT091001 can improve visual function in subjects with LCA or RP due to RPE65 or LRAT mutations

• to evaluate duration of visual function improvement (if observed)

Clinical Trial Description

n/a

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• LCA (Leber Congenital Amaurosis)
• Leber Congenital Amaurosis
• Retinitis
• Retinitis Pigmentosa
• RP (Retinitis Pigmentosa)

• NCT number: NCT01014052
• Study type: Interventional
• Source: QLT Inc.
• Status: Completed
• Phase: Phase 1
• Start date: November 2009
• Completion date: August 2012