Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis

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

Timeline

Start

2021-01-11

Primary completion

2021-10-27

Completion

2021-12-29

Outcome

Outcome not reported

Stopped: “Big delay on study activation (approval procedures by IRBs, hospitals contract negotiation) and on enrolment deadline due to CoViD-19; Placebo stock expires November 2021 and new batch not be available for at least one year”

Drugs

Indications