Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease

• Status: Terminated
• Trial end date: 2009-12-14
• Target enrollment: 0
• Participant gender: All

Summary

The main purpose of this study was to determine the safety and tolerability of 3 different doses of duvoglustat (AT2220) in participants affected by Pompe disease. The study also evaluated the effects of duvoglustat on functional parameters in Pompe disease.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amicus Therapeutics
• Treatments: 1-Deoxynojirimycin

Criteria

Inclusion Criteria:

• Male or female, 18 to 74 years of age inclusive

• Diagnosis of Pompe disease based on clinical assessment, enzyme assay, and/or genotyping. Confirmatory genotyping will be performed on all participants who are screened for the study

• Naïve to enzyme replacement therapy (ERT) or has not received ERT in the 3 months prior to screening

• Willing not to initiate ERT or other prohibited treatment during study participation

• Functional grade for arms and/or legs ≥2 OR sitting forced vital capacity ≥30% and <80% of predicted value, reproducible between screening and baseline (±15%)

• Participants of reproductive potential agree to use reliable methods of contraception during the study

• Participant or legal representative is willing and able to provide written informed consent

Exclusion Criteria:

• Any intercurrent condition that may preclude accurate interpretation of study data

• Obstructive pulmonary disease

• Invasive ventilatory support

• Use of noninvasive ventilatory support >8 hours/day while awake

• History of QTc prolongation >450 milliseconds (msec) for males and >470 msec for females

• History of allergy or sensitivity to the study drug, including any prior serious adverse reaction to iminosugars (such as miglustat or miglitol)

• Pregnancy or breast-feeding

• Current or recent drug or alcohol abuse

• Treatment with another investigational drug within 30 days of study start

• Use of prohibited medications ≤3 months prior to screening

• Otherwise unsuitable for the study in the opinion of the Investigator