6 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…
The aim is to assess the long-term safety of topical use of ZORBLISA in patients with Epidermolysis Bullosa (EB).
The primary objective is to compare the efficacy and safety of SD-101-6.0vs. SD- 101-0.0 (placebo) in patients with Simplex, RecessiveDystrophic, or Junctional non Herlitz Epidermolysis Bullosa.The primary endpoint is the complete closure of the…
Primary ObjectivesTo evaluate the safety and tolerability of AP30663 in healthy males at doses up to 12mg/kgExploratory ObjectiveTo evaluate the effect of AP30663 on electrocardiographical parameters.
Primary Objective- To evaluate the safety and tolerability of AP30663 in healthy malesSecondary Objectives- To evaluate the pharmacokinetic profile of AP30663.Exploratory Objectives- To evaluate the effect of AP30663 on electrocardiographical…