New hope for kids with pompe: enzyme combo trial underway
NCT ID NCT03911505
First seen Sep 30, 2025 · Last updated May 15, 2026 · Updated 31 times
Summary
This study tests a combination of two medicines (cipaglucosidase alfa and miglustat) in children aged 0 to 18 with late-onset Pompe disease, a rare genetic disorder that causes muscle weakness. The goal is to see if the treatment is safe and works better than current enzyme replacement therapy. About 21 children who have either never received treatment or have been on standard therapy will take part.
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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
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