A Phase 2a study of AT2220 with ERT in Subjects with Pompe Disease

• Research type

  Research Study

• Full title

  An Open-Label, Multi-Center, Study to Investigate Drug-Drug Interactions Between AT2220 (Duvoglustat Hydrochloride) and Alglucosidase Alfa in Patients with Pompe Disease

• IRAS ID

  89092

• Contact name

  Mark Roberts

• Sponsor organisation

  Amicus Therapeutics Inc

• Eudract number

  2011-002154-32

• Clinicaltrials.gov Identifier

  NCT01380743

• Research summary

  This is a phase 2a, open-label, single dose study funded by Amicus Therapeutics. The research study is being conducted to test if AT2220 can be safely used in combination with enzyme replacement therapy (ERT) in patients with Pompe disease. This study will look at the safety and effects of ERT when given with AT2220. This study will also examine the reaction of the subjects’ body to ERT with and without AT2220. This information may help doctors learn how to treat patients with AT2220 and ERT together. It will also show if the improvement in ERT enzyme levels, when taken with AT2220, should be studied further for treatment of patients with Pompe disease. Approximately 16 patients with Pompe disease on a stable dose of alglucosidase alfa for at least 3 months before screening will be recruited in this study in 4 countries. Approximately 4 participants will be recruited in the UK. The duration of participation could be up to 3 months. The study involves a screening period, 2 treatment periods and a follow up visit. During the first treatment period all participants will receive their standard enzyme replacement therapy (ERT). During the second treatment period participants will receive a dose of the study drug AT2220 1 hour before their standard ERT. There are 4 treatment groups consisting of ascending doses of AT2220. The participants’ assignment to treatment group will depend on when they enter the study. This study involves procedures including:- Physical examination, vital signs, ECG, blood tests, urine tests, muscle strength tests and muscle biopsies.

• REC name

  North West - Liverpool Central Research Ethics Committee

• REC reference

  11/NW/0745

• Date of REC Opinion

  7 Dec 2011

• REC opinion

  Further Information Favourable Opinion