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

Completed · Phase 1

Conditions studied: LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)

In brief

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)

Key facts

Study ID

NCT01014052

Run by

QLT Inc.

People needed

32

Starts

2009-11-01

Expected to finish

2012-08-01

Last updated by the study team

2013-05-14

Who can join

Age: 5 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Subjects diagnosed with LCA or RP (with a mutation in either RPE65 or LRAT)
• Subjects with LCA must be 5-65 years of age
• Subjects with RP must be 18-65 years of age
• Subjects who have a "best-corrected" visual acuity of 3 letters or better (20/800 Snellen equivalent) or viable photoreceptors on OCT/FAF.

You may not qualify if…

• Subjects who are actively participating in an experimental therapy study or who have received experimental therapy within 60 days of Day 0.
• Subjects with any clinically important abnormal physical finding at Screening.
• Subjects who have taken any prescription or investigational oral retinoid medication (e.g., Accutane,® Soriatane®) within 6 months of Day 0 and subjects who did not tolerate their previous oral retinoid medication will be excluded regardless of the time of last exposure.
• Subjects with a history of diabetes or chronic hyperlipidemia, hepatitis, pancreatitis, or cirrhosis.
• Subjects who have taken any supplements containing ≥10,000 IU vitamin A within 60 days of screening

Where it is running

• The Chicago Lighthouse for People Who Are Blind or Visually Impaired (The Pangere Center For Inherited Retinal Diseases) — Chicago, Illinois, United States
• Wilmer Eye Institute (Johns Hopkins University) — Baltimore, Maryland, United States
• Scheie Eye Institute — Philadelphia, Pennsylvania, United States
• Montreal Children's Hospital, McGill University Health Centre — Montreal, Quebec, Canada
• Institute for Ophthalmic Research, University of Tubingen — Tübingen, Germany
• The Rotterdam Eye Hospital — Rotterdam, Netherlands
• Moorefield Eye Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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