3 results
3. STUDY OBJECTIVES3.1. Primary ObjectivesThe primary objectives are:• To determine if CAEL-101 and treatment for PCD improves overall survival in Mayo stage IIIa AL amyloidosis patients who are treatment naïve compared to treatment for PCD alone•…
3. STUDY OBJECTIVES3.1. Primary ObjectivesThe primary objectives are:• To determine if CAEL-101 and treatment for PCD improves overall survival in Mayo stage IIIb AL amyloidosis patients who are treatment naïve compared to treatment for PCD alone•…
Primary: To assess the efficacy of GNX compared with PBO, as adjunctive therapy for the treatment of primary seizure types in children with genetically-confirmed PCDH19-related epilepsy during the 17-week double-blind (DB) phase. Secondary: • To…