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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Locations
-
Duke University Medical Center
RECRUITINGDurham, North Carolina, 27710, United States
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