Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis
Status:
Withdrawn
Trial end date:
2021-12-29
Target enrollment:
Participant gender:
Summary
Randomized, double-blind, placebo-controlled, parallel group study where subjects will
receive velmanase alfa or placebo for 24 weeks.
Each subject undergoes to 8 complete visits at the clinic for clinical, laboratory and
functional assessments. Study treatment is administered weekly through i.v. infusions