New hope for rare disease: experimental drug targets PMM2-CDG

NCT ID NCT05549219

Completed Disease control Sponsor: Glycomine, Inc. Source: ClinicalTrials.gov ↗

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

Summary

This study tested an experimental drug called GLM101 in 27 people with PMM2-CDG, a rare genetic condition that causes problems with balance and movement. Participants received different doses of the drug intravenously over 24 weeks. The main goal was to see if the drug improves coordination and to check its safety.

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Conditions

The condition(s) this trial relates to.

PMM2-congenital disorder of glycosylation

As listed by the trial registrant

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

PHOSPHOMANNOMUTASE 2 DEFICIENCY PMM2-CDG

More trials for these conditions

Other studies related to the condition(s) this trial covers.

Contacts and locations

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Locations

Clinical Research of West Florida

Tampa, Florida, 33606, United States
Great Ormond Street Hospital

London, WC1N3JH, United Kingdom
Hospital Sant Joan de Déu

Barcelona, 08950, Spain
Mayo Clinic

Rochester, Minnesota, 55905, United States