3 results
• To evaluate whether 7-day treatment with oral QLT091001 can improve visual function in subjects with LCA or RP caused by RPE65 or LRAT gene mutations.• To evaluate duration of visual function improvement (if observed) in subjects with LCA or RP…
The study objectives are:- to characterize the biotransformation pathways, the routes and rates of excretion, and total recovery of the 14C-labeled research medication and its radiolabeled metabolites- to characterize the pharmacokinetics (PK) of…
Safety:* Evaluate the safety of AVXS-101 through incidence of adverse events (AEs) and/or serious adverse events (SAEs)* Evaluate the safety of AVXS-101 based on the change from baseline in clinical laboratory parametersEfficacy objectives will be…