New hope for kids with pompe: enzyme combo trial underway
NCT ID NCT03911505
First seen Sep 30, 2025 · Last updated May 23, 2026 · Updated 32 times
Summary
This study tests a new treatment (cipaglucosidase alfa plus miglustat) for children aged 0 to 18 with late-onset Pompe disease. It aims to check safety, how the drug moves through the body, and its effects. Participants may have received prior enzyme therapy or be new to treatment.
Disclaimer
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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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Contacts and locations
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Locations
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Duke University Medical Center
Durham, North Carolina, 27710, United States
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Gunma University Hospital
Gunma, 371-8511, Japan
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Izumi City General Hospital
Osaka, Izumi-Shi, 594-0073, Japan
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Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax, Virginia, 22030, United States
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San Gerardo Hospital
Monza, 20900, Italy
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SphinCS GmbH Clinical Science for LSD
Hochheim am Main, Hesse, 65239, Germany
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St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
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Tohoku University Hospital
Miyagi, 980-8574, Japan
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Tokyo Women's Medical University
Tokyo, 162-8666, Japan
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UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
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University of Calgary
Calgary, Alberta, T3B 6A8, Canada
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University of Florida Clinical Research Center
Gainesville, Florida, 32610, United States
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University of Utah, Clinical and Translational Sciences Institute
Salt Lake City, Utah, 84108, United States
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Wolfson Children's Hospital
Jacksonville, Florida, 32207, United States
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Women's and Children's Hospital
North Adelaide, South Australia, 5006, Australia
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Woodruff Memorial Research Building
Atlanta, Georgia, 30322, United States
Conditions
Explore the condition pages connected to this study.