New hope for kids with rare Muscle-Weakening disease
NCT ID NCT03911505
Summary
This study is testing a new combination drug (cipaglucosidase alfa with miglustat) for children with late-onset Pompe disease, a rare genetic disorder that weakens muscles. The main goal is to see if the treatment is safe and how well it works in children from birth up to 18 years old. It is for kids who have never had enzyme replacement therapy before, as well as those who are already receiving a standard treatment.
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.