Desperate pompe patients get early access to experimental drug combo

NCT ID NCT03865836

AVAILABLE Disease control Sponsor: Amicus Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This expanded access program provides ATB200/AT2221 to people with Pompe disease who cannot join ongoing clinical trials or choose not to use standard therapy. The treatment combines an enzyme replacement therapy with a chaperone drug to help break down glycogen buildup. Participants must have a confirmed diagnosis and agree to share their health data.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: ATB200 (enzyme replacement therapy) and AT2221 (chaperone drug)
What this could lead to: If this treatment works, it could offer a new option for managing Pompe disease, potentially improving muscle function and survival.
What could go wrong: This is an early access program, not a formal trial, so effectiveness and safety are still being studied. Benefits are uncertain, and there may be unknown side effects.

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.

glycogen storage disease II Glycogen Storage Disease Type II

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

POMPE DISEASE

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

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