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)
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
32
oral QLT091001 administered once daily for 7 days
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
Visual Field
Time frame: 12 months
Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events
Time frame: 12 months
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