5 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…
Primary: To estimate the antitumor activity (assessed as overall response rate) of PDR001 as a single agent in patients with non-functional neuro-endocrine tumors (NET).Secondary: To estimate efficacy (duration of response) of PDR001. Safety and…
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…
* Safety Run inTo determine the recommended regimen of PDR001 in combination with dabrafenib and trametinib for the randomized part (part 3)* Biomarker cohortTo evaluate changes in the immune microenvironment and biomarker modulations upon treatment…