5 results
The overall objective of this study is to evaluate the efficacy and safety of neoGAA in treatment-naïve patients with LOPD as compared to alglucosidase alfa, when this is administered biweekly for a period of 49 weeks. Also, there is an open-label…
Main objectives Part 1• To evaluate the safety and tolerability of a single oral dose of HTL0018318, administered at one of at least three ascending dose levels (maximum of 8 ascending dose levels), in healthy younger adult male subjects.• To…
1. To evaluate the safety and tolerability of ascending multiple oral doses of HTL18318 in healthy adult male and female subjects.2. To evaluate the pharmacokinetics of ascending multiple oral doses of HTL18318 in healthy adult male and female…
1. Primary Objectives• To evaluate the safety and tolerability of VX 440 monotherapy and VX 440 in dual and triple combination with VX-661 and IVA• To evaluate the efficacy of VX 440 monotherapy and VX 440 in dual and triple combination with VX 661…
* To evaluate the safety and tolerability of adding multiple oral doses of HTL0018318 in elderly volunteers taking donepezil at steady state.* To compare the pharmacokinetic profiles of HTL0018318 and donepezil when given alone and in combination at…