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 range
18 Years – 74 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Proportion Of Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs)