New hope for kids with pompe: enzyme combo trial underway
NCT ID NCT03911505
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new treatment called cipaglucosidase alfa/miglustat in children (0 to under 18 years) with late-onset Pompe disease. The goal is to see if it is safe and how well it works. Participants either have never had enzyme therapy before or have been on it for at least 6 months. The treatment aims to control the disease, not cure it.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for POMPE DISEASE LATE-ONSET are added.
By submitting, you agree to our Terms of use
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
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
-
Duke University Medical Center
Durham, North Carolina, 27710, United States
-
Gunma University Hospital
Gunma, 371-8511, Japan
-
Izumi City General Hospital
Osaka, Izumi-Shi, 594-0073, Japan
-
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax, Virginia, 22030, United States
-
San Gerardo Hospital
Monza, 20900, Italy
-
SphinCS GmbH Clinical Science for LSD
Hochheim am Main, Hesse, 65239, Germany
-
St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
-
Tohoku University Hospital
Miyagi, 980-8574, Japan
-
Tokyo Women's Medical University
Tokyo, 162-8666, Japan
-
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
-
University of Calgary
Calgary, Alberta, T3B 6A8, Canada
-
University of Florida Clinical Research Center
Gainesville, Florida, 32610, United States
-
University of Utah, Clinical and Translational Sciences Institute
Salt Lake City, Utah, 84108, United States
-
Wolfson Children's Hospital
Jacksonville, Florida, 32207, United States
-
Women's and Children's Hospital
North Adelaide, South Australia, 5006, Australia
-
Woodruff Memorial Research Building
Atlanta, Georgia, 30322, United States