7 results
Primary objective: • To assess the efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 24 weeks in ambulant subjects with DMD.Secondary objectives:• To assess the safety and tolerability of 2 different dosing…
The objectives of the study are to: -To evaluate the safety and tolerability of MOTREM in patients with septic shock. -To evaluate the effects of MOTREM exposure over up to 5 days in patients with septic shock -To evaluate the PK/PD and dose/PD…
Primary objective:Evaluate the safety, tolerability, pharmacokinetics (PK), and immunogenicity of multiple doses of intravenous (IV) ATYR1940 in adults 18 to 65 years of age, inclusive, with FSHDSecondary objective:Explore pharmacodynamic (PD)…
The primary objective of the study is to evaluate the safety, tolerability, PK, and PD of AL002 administered in single ascending doses in healthy participants and multiple doses in participants with mild to moderate AD.
Primary:To determine the efficacy of INCB000928 for the prevention of new HO in participants with FOP.Key Secondary:To further evaluate the efficacy of INCB000928 in the reduction of flares and improvement in flare-related symptoms.Secondary:- To…
To determine RD of BLU-263To assess if treatment with BLU-263 improves outcomes compared to placebo + BSC, as assessed using the ISM-SAFTo assess the long-term safety and tolerability of treatment with BLU-263To assess the long-term efficacy of…
To evaluate the efficacy of AL002 in participants with Early AD in delaying disease progression compared to standard of care