To Assess AT2101 in Adult Patients with Type 1 Gaucher Disease.

• Research type

  Research Study

• Full title

  An Open-Label Study to Assess the Pharmacokinetics, Safety, and Pharmacodynamics of repeated doses of orally administered AT2101 in adult patients with Type 1 Gaucher Disease.

• IRAS ID

  17408

• Sponsor organisation

  Amicus Therapeutics, Inc.

• Eudract number

  2009-009329-27

• ISRCTN Number

  No number provided

• Clinicaltrials.gov Identifier

  No number provided

• Research summary

  AT2101 is a drug that may be useful in the treatment Gaucher disease. The purpose of this study is to examine the safety of the drug, the effect on the body and the way the body handles (absorbs, distributes, breaks down and excretes) multiple oral doses of AT2101 in adult volunteers with type 1 Gaucher disease. This is an inherited illness caused by the lack of an enzyme called b-glucosidase that the body needs to break down a particular kind of fat called glucocerebroside. This study involves 6-8 adult patients with type 1 Gaucher disease who are currently receiving enzyme replacement therapy (ERT) with imiglucerase. Each volunteer will be admitted to the clinical unit on two occasions for two nights and three days. This will be followed by an outpatient follow up visit 5 to 7 days after last dosing.

• REC name

  London - London Bridge Research Ethics Committee

• REC reference

  09/H0804/3

• Date of REC Opinion

  27 Jul 2009

• REC opinion

  Further Information Favourable Opinion