Experimental drug GLM101 targets rare PMM2-CDG in pivotal trial
NCT ID NCT06892288
First seen Jun 25, 2026 · Last updated Aug 14, 2026 · Updated 2 times
Summary
This study tests a drug called GLM101 for people with PMM2-CDG, a rare inherited disease that affects movement and coordination. About 50 children and adults will receive weekly infusions of either GLM101 or a placebo for 24 weeks, followed by an open-label phase where everyone gets the drug. The main goal is to see if GLM101 improves ataxia (loss of muscle control) and other motor functions.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- GLM101 (a drug given as a weekly intravenous infusion)
- What this could lead to
- If it works, this could point toward a treatment that improves coordination and movement in people with PMM2-CDG, a rare and serious genetic disease.
- What could go wrong
- This is a mid-stage trial with only 50 participants, so results may not apply to everyone. The drug is still experimental, and its long-term safety and effectiveness are not yet proven.
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
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AP-HP Hopital Necker-Enfants Malades
Paris, 75015, France
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Azienda Ospedaliero Universitaria Pisana
Pisa, 56126, Italy
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Azienda Ospedaliero Universitaria Policlinico G. Rodolico-San Marco
Catania, 95124, Italy
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Birmingham Women's and Children's NHS Foundation Trust
Birmingham, B4 6NH, United Kingdom
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Hospital Sant Joan de Déu
Esplugues de Llobregat, 08950, Spain
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Hospital Universitario 12 de Octubre
Madrid, 28041, Spain
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Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
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Instytut Matki i Dziecka
Warsaw, 01-211, Poland
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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UZ Leuven, Campus Gasthuisberg
Leuven, 3000, Belgium
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Unidade Local de Saúde de Santo António
Porto, 4099-001, Portugal
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Universitaetsklinikum Muenster
Münster, 48149, Germany
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University of Minnesota
Minneapolis, Minnesota, 55455, United States
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Vseobecna fakultni nemocnice v Praze
Prague, 2, 128 0, Czechia
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