New pompe disease drug moves to first human tests

NCT ID NCT06109948

Completed Disease control Sponsor: Aro Biotherapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase study tested a new drug called ABX1100 in 46 people, including healthy volunteers and patients with late-onset Pompe disease. The main goal was to check safety and how the drug moves through the body. Researchers gave single or multiple doses to find the right amount for future studies.

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Conditions

The condition(s) this trial relates to.

glycogen storage disease due to acid maltase deficiency, late-onset

As listed by the trial registrant

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

HEALTHY LATE-ONSET POMPE DISEASE

Contacts and locations

Locations

Lysosomal and Rare Disorders Research and Treatment Center, Inc

Fairfax, Virginia, 22030, United States
MAGIC clinic

Calgary, Alberta, Canada
McMaster University

Hamilton, Ontario, Canada
UCI

Orange, California, 92868, United States
Washington University School of Medicine

St Louis, Missouri, 63110, United States

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