6 results
Primary:To determine the efficacy of AG10 in the treatment of subjects with symptomatic transthyretin amyloid polyneuropathy (ATTR-PN) by evaluating the difference between the AG10 and placebo groups in Modified Neuropathy Impairment Score + 7 (mNIS…
We hypothesized that bowel preparation will be comparable between groups as measured using the Boston Bowel Preparation Scale and that the adenoma detection rate will be comparable across groups. Furthermore, we hypothesized that patient experience…
This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of crovalimab compared with placebo as adjunct treatment for the prevention of vaso-occlusive episodes (VOEs) in patients with sickle cell disease (SCD).
Key PrimaryPart A• To determine the efficacy of acoramidis (AG10) in the treatment of subjects with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM) by evaluating the difference between the acoramidis and placebo groups in the change from…
Primary• To assess safety and tolerability of acoramidis in participants with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM)Secondary• To evaluate the effect of acoramidis on all-cause mortality and cardiovascular (CV) mortality• To…
This study will evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of crovalimab compared with placebo for the management of acute uncomplicated vaso-occlusive episodes (VOE) in patients with sickle cell disease (SCD).