NCT01014052

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

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
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 32 (actual)
• Sponsor: QLT Inc. · Industry
• Sex: All
• Age: 5 Years – 65 Years
• Healthy volunteers: Not accepted

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)

Conditions

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

Interventions

Type	Name	Description
DRUG	QLT091001	oral QLT091001 administered once daily for 7 days

Timeline

• Start date: 2009-11-01
• Primary completion: 2012-08-01
• Completion: 2012-08-01
• First posted: 2009-11-16
• Last updated: 2013-05-14

Locations

7 sites across 5 countries: United States, Canada, Germany, Netherlands, United Kingdom