Experimental drug GLM101 targets rare PMM2-CDG in pivotal trial
NCT ID NCT06892288
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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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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