Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease
Status:
Completed
Trial end date:
2017-10-09
Target enrollment:
Participant gender:
Summary
Six patients with Fabry disease will be recruited. Patients will receive a single dose of 0.2
mg/kg recombinant human alpha-galactosidase A produced in moss (moss-aGal) as intravenous
infusion. Patients will be hospitalized during the infusion and for at least 24 hours after
the end of the infusion. Treatment will be administered sequentially: if a patient shows no
safety concerns on the treatment day, treatment of the next patient will commence on the
following day.