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
- Conditions
- LCA (Leber Congenital Amaurosis)RP (Retinitis Pigmentosa)
- Registration Number
- NCT01014052
- Lead Sponsor
- QLT Inc.
- Brief 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)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 32
-
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.
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method Visual Field 12 months
- Secondary Outcome Measures
Name Time Method Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events 12 months
Trial Locations
- Locations (7)
Institute for Ophthalmic Research, University of Tubingen
🇩🇪Tubingen, Germany
Montreal Children's Hospital, McGill University Health Centre
🇨🇦Montreal, Quebec, Canada
Moorefield Eye Hospital
🇬🇧London, United Kingdom
Wilmer Eye Institute (Johns Hopkins University)
🇺🇸Baltimore, Maryland, United States
Scheie Eye Institute
🇺🇸Philadelphia, Pennsylvania, United States
The Chicago Lighthouse for People Who Are Blind or Visually Impaired (The Pangere Center For Inherited Retinal Diseases)
🇺🇸Chicago, Illinois, United States
The Rotterdam Eye Hospital
🇳🇱Rotterdam, Netherlands