6 results
Primary end points:Safety (>1 toxic death per arm) and tolerability (relevant grade 4 toxicities) in not more than 33% of patients for the three treatment arms.Secondary end points:* Reduction of grade 2-4 key side effects in the experimental…
This multicentre phase III study is designed to assess the efficacy and safety of recombinant versus E-Coli derived Aaparaginase from Medac, during treatment of children with newly diagnosed ALL according to the DCOG ALL-10 protocol.Futhermore: To…
This non-controlled multicentric phase II study is designed to assess the safety and to describe (in relation to children of higher age) the pharmacodynamics of recombinant ASNase for first-line treatment of infants (< 1 year of age at…
Primary Objective:To demonstrate the efficacy of repeated daily doses of 120 µg/kg/day odevixibat in relieving pruritus in patients with ALGS.Secondary Objectives:To assess the impact of odevixibat on serum bile acid levels in patients with ALGS.To…
Primary Objective:The primary objective is to evaluate the efficacy of repeated once-daily doses of odevixibat versus placebo in children with biliary atresia (BA) post Kasai hepatoportoenterostomy(HPE) based native liver survival (NLS) of up to 104…
To demonstrate a sustained effect of odevixibat on pruritus in patients with ALGS who have completed study A4250-012 (ASSERT)