Pompe disease study peers into the brain for hidden clues
NCT ID NCT04639336
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study aims to learn how Pompe disease affects the brain and nerves over the long term. Researchers will use brain scans, thinking and language tests, and muscle checks in 45 people with Pompe disease. Participants will be followed for 3 to 6 years. No new treatment is being tested; the goal is to gather knowledge to improve future care.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- What this could lead to
- If successful, this study could reveal how Pompe disease affects the brain and nerves over time, guiding future care and monitoring.
- What could go wrong
- This is an observational study, not a treatment trial. It may not lead to direct medical changes, and results depend on a small group of 45 participants.
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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
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Duke University Medical Center
RECRUITINGDurham, North Carolina, 27710, United States
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