5 results
To demonstrate the efficacy of repeated daily doses of 40 µg/kg/day and 120 µg/kg/day A4250 in children with progressive familial intrahepatic cholestasis (PFIC) Types 1 and 2, as determined by the following:* Proportion of patients experiencing at…
Primary objective: To determine the efficacy of the combination of acalabrutinib and venetoclax without obinutuzumab (AV; Arm A), or with obinutuzumab (AVG; Arm B) compared with chemoimmunotherapy (fludarabine/cyclophosphamide/rituximab [FCR]/…
The aim is thereby to reduce the risk of serious infections and the need for regular CLL treatment.
Therapy:The investigational product, ACP-196, will be supplied as hardgelatin capsules for oral administration.Commercially available ibrutinib (IMBRUVICA®) will be used asthe reference therapy.Objectives: Primary Objective:To assess whether ACP-196…
Primary Objective:To evaluate the effect of sonlicromanol on motor symptom severity in children with genetically confirmed mitochondrial disease affecting oxidative phosphorylation during a 6 month treatment period (GMFM).