Randomized, controlled trial of miglustat in Gaucher's disease type 3.

ABSTRACT:

Objective

To evaluate the efficacy and safety of miglustat, concomitant with enzyme replacement therapy (ERT), in patients with Gaucher's disease type 3 (GD3).

Methods

This 24-month, phase II, open-label clinical trial of miglustat in GD3 was conducted in two phases. During the initial 12 months, patients were randomized 2:1 to receive miglustat or "no miglustat treatment." The randomized phase was followed by an optional 12-month extension phase in which all patients received miglustat. All patients received ERT during the 24-month period. The primary efficacy end points were change from baseline to months 12 and 24 in vertical saccadic eye movement velocity as determined by the peak amplitude versus amplitude regression line slope. Secondary end points included changes in

SUBMITTER: Schiffmann R 

PROVIDER: S-EPMC2605167 | biostudies-literature | 2008 Nov

REPOSITORIES: biostudies-literature