8 results
The purpose of this study is to compare the efficacy and the safety of an experimental drug, masitinib,in combination with riluzole to placebo in combination with riluzole administered during 48 weeks to patients suffering from ALS.You will be…
The objective is to compare the efficacy and safety of masitinib 12 mg/kg/day to sinutinib 50 mg/day
The objective of the study is to compare the safety and efficacy of masitinib at 4.5 mg/kg/day or masitinib at 4.5 mg/kg/day with a dose escalation to 6 mg/kg/day after three month of treatment versus placebo in the treatment of patients with…
- To assess the pharmacokinetic profile of cysteamine base after single dose administration of the new sustained-release cysteamine bitartrate (PO-001) to healthy volunteers, in comparison with Cystagon® and Procysbi®.- To collect safety/…
To evaluate the efficacy and safety of oral masitinib versus placebo in the treatment of patients suffering from Smouldering or Indolent SevereSystemic mastocytosis with handicap unresponsive to optimal symptomatic treatment.
The primary objectives are: To evaluate the CNS activity of DNL310 vs idursulfase as measured by the cerebrospinal fluid (CSF) concentration of heparan sulfate (HS) in participants with the neuronopathic form of mucopolysaccharidosis type II (nMPS…
The primary objective of the study is the following:• To characterize the safety and tolerability of DNL310 in pediatric subjects with MPS II (all cohorts)The secondary objectives of the study are as follows:• To characterize the CNS effects of…
The objective is to evaluate the efficacy and safety of two doses of masitinib (4.5 mg/kg/day and 6.0 mg/kg/day) versus matching placebo in patients diagnosed with ALS treated with Riluzole (50 mg bid).The primary objective is to demonstrate…