Hope for pompe patients: could a switch in enzyme therapy slow decline?
NCT ID NCT07652814
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether switching to avalglucosidase alfa is safe and more effective for people with late-onset Pompe disease whose condition has worsened on the standard treatment, alglucosidase alfa. Participants receive biweekly infusions of the new drug and are monitored for muscle strength, lung function, and quality of life. The trial includes about 6 people aged 5 to 55 in the Netherlands.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- avalglucosidase alfa
- What this could lead to
- If successful, this could offer a better treatment option for people with late-onset Pompe disease whose condition declines on current enzyme therapy.
- What could go wrong
- This is a small, early-phase study with only 6 participants, so results may not apply to everyone. The drug may not work better or could cause side effects like infusion reactions.
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
-
Erasmus MC
Rotterdam, 3015 GD, Netherlands
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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