New drug combo shows promise for rare pompe disease
NCT ID NCT02675465
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested whether a new combination of two drugs (ATB200 and AT2221) is safe for adults with Pompe disease, a rare genetic disorder that causes muscle weakness. 29 participants received the drugs through an IV and by mouth. The main goal was to check for side effects and see how the body processes the medication.
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Abramson Cancer Center Chester County Hospital
West Chester, Pennsylvania, 19380, United States
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Duke University Medical Center
Durham, North Carolina, 27710, United States
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Emory University Division of Medical Genetics
Decatur, Georgia, 30033, United States
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Erasmus Medical Center
Rotterdam, Netherlands
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Friedrich-Baur-Institure, Dep of Neurology - University Munich
Munich, 80336, Germany
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Great Falls Clinic, LLP
Great Falls, Montana, 59405, United States
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Infusion Associates
Grand Rapids, Michigan, 49525, United States
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Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)
Fairfax, Virginia, 22030, United States
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Neuromuscular Research Centre
Phoenix, Arizona, 85028, United States
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Perelman Center for Advanced Medicine
Philadelphia, Pennsylvania, 19104, United States
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Rutgers New Jersey Medical School
Newark, New Jersey, 08103, United States
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Salford Royal NHS Foundation Trust
Salford, M6 8HD, United Kingdom
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School of Medicine, University of Auckland
Auckland, 01051, New Zealand
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University Children's Hospital Department of Neuropediatrics and Inborn Metabolic Disorders, St. Josefs-Hospital
Bochum, 44791, Germany
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University Hospital Birmingham NHS Foundation Trust, Queen Elizabeth Medical Center
Birmingham, B15 2TH, United Kingdom
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University of California Irvine
Orange, California, 92868, United States
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University of Florida
Gainesville, Florida, 32610, United States
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University of Pittsburgh
Pittsburgh, Pennsylvania, 15213, United States
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Womens & Childrens Hospital, Adelaide
North Adelaide, South Australia, 05006, Australia