Pompe disease study aims to unlock immune secrets for better treatment
NCT ID NCT01665326
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study follows up to 400 children with Pompe disease to see how their immune system reacts to enzyme replacement therapy. Researchers will collect medical records from birth to age 18 to understand which children develop antibodies that block treatment. The goal is to improve care by identifying who might benefit from immune-suppressing drugs alongside standard therapy.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
alglucosidase alfa (Myozyme/Lumizyme)
What this could lead to
If successful, this study could help doctors predict which children with Pompe disease will respond best to enzyme therapy and who might need immune suppression.
What could go wrong
This is an observational study, not a treatment trial. It will not directly test a new therapy, so any benefits are indirect and long-term.
Disclaimer
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This is a summary of
the original study
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Duke University Medical Center
RECRUITINGDurham, North Carolina, 27710, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact