6 results
To determine whether doubling the dose of corticosteroid injections in the treatment of arthritis in knee joints is more effective in the relief of symptoms as measured by change in the assessment of arthritis burden by the patient on a Likert 5-…
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…
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…
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…