Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease
Status:
Completed
Trial end date:
2021-03-18
Target enrollment:
Participant gender:
Summary
During the past two years, the investigator has performed succsefully an IIR wherein patients
with GD, previously treated with velaglucerase alfa ERT were gradually switched to a 10
minutes (rapid) administration of the same ERT. The success was expressed as safety (no
clinically meaningful AEs, no antibodies detected, home therapy), efficacy ("lack of
deterioration") and patients' satisfaction. The latter was based not just on specific
questionnaires and analog scales, but particularly by the patients' sharing the experience
with other patients and consequently repeated requests by many to switch to a rapid
administration of their ERT.
Therefore, the investigator is hereby proposing to investigate the safety and efficacy of a
10 minutes administration of velaglucerase alfa in a cohort of treatment-naive patients.
The current VPRIV label is restricted to a dosage of 60 units/kg body weight every other week
(60 units/kg EOW) - this dose will be used throughout the study period. The enzyme will be
provided by Shire, which will also provide a research grant for the conduction of the trial.