Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease (NCT00688597) | Clinical Trial Compass
TerminatedPhase 2
Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
Stopped: Per protocol, 3 sequential dose cohorts were planned. Study discontinued by Sponsor based upon serious adverse events in first 2 of 3 participants in Cohort 1.
United States3 participantsStarted 2008-12-08
Plain-language 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.
Who can participate
Age range18 Years – 74 Years
SexALL
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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
* Other…
What they're measuring
1
Proportion Of Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs)