4 results
Primary objective: Assessment of the value of the short-term (2 weeks) response of IGF-I (peak IGF-I SDS) to GH in a dosage of 1.4 mg/m2/day (as part of a dose-escalation IGF-I generation test) in comparison to baseline IGF-I to predict the 1 year…
This is a follow-up (FU) study for subjects who completed Study ALX0681-C301 (HERCULES) according to the protocol (i.e., completed the Final [28 day] FU visit). Subjects who completed Study ALX0681-C301 will be given the option to participate in…
Currently, caplacizumab is being developed for treatment of acquired thrombotic thrombocytopenic purpura (TTP). TTP is a rare and potentially life-threatening thrombotic microangiopathy, in which accumulation of ULvWF multimers leads to an increased…
Safety (Primary) ObjectiveThe primary objective for this study is as follows:- To evaluate the safety and tolerability of cobimetinib in children and young adults, including estimation of the maximum-tolerated dose (MTD) or the maximum administered…