3 results
PART I:Primary:• To investigate the safety and tolerability of two doses of VAD044 administered daily for up to 12 weeks in HHT patients.Secondary:• To assess the effects of two doses of VAD044 for up to 12 weeks of treatment on:o Epistaxis:•…
Primary objective: To evaluate the effect of navitoclax in combination with ruxolitinib on splenomegaly response when compared to ruxolitinib in subjects with myelofibrosis.Secondary objectives:• To evaluate the effect of navitoclax in combination…
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…