Status:
TERMINATED
Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
Lead Sponsor:
Amicus Therapeutics
Conditions:
Pompe Disease
Eligibility:
All Genders
18-74 years
Phase:
PHASE2
Brief 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...
Detailed Description
This was a Phase 2, open-label study in participants with Pompe disease, a lysosomal storage disorder. Duvoglustat is designed to act as a pharmacological chaperone of alpha-glucosidase, in order to r...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
December 8 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 14 2009
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT00688597
Start Date
December 8 2008
End Date
December 14 2009
Last Update
September 11 2025
Active Locations (1)
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1
Decatur, Georgia, United States, 30033