Hope for rare disease: new drug shows promise in Long-Term study
NCT ID NCT06657859
First seen Jun 27, 2026 · Last updated Jul 29, 2026 · Updated 6 times
Summary
This study is for people with PMM2-CDG, a rare genetic disorder, who have already taken the experimental drug GLM101 in a previous trial. The goal is to see if GLM101 is safe and effective over a longer period. Participants will receive weekly infusions of GLM101 at the same dose as before. The study will monitor side effects and changes in movement and coordination.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- GLM101 (given intravenously)
- What this could lead to
- If successful, this could show that GLM101 is safe and helps control symptoms of PMM2-CDG over the long term, offering a treatment option for this rare disease.
- What could go wrong
- This is an early-phase, open-label extension study with no placebo group, so results may be less reliable. The drug may still cause side effects or not work as hoped.
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 Universitaire 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 - Presidio Ospedaliero G. Rodolico
Catania, 95124, Italy
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Birmingham Children's Hospital
Birmingham, B4 6NH, United Kingdom
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Great Ormond Street Hospital for Children
London, WC1N 3JH, 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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Hospital Universitario 12 de Octubre - Unidad Pediatrica de Investigacion y Ensayos Clinicos (UPIC)
Madrid, 28041, Spain
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, 19104, United States
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The Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
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Unidade Local de Saúde de Santo António, E.P.E
Porto, 4099-001, Portugal
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Universitaetsklinikum Münster
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, 128 08, Czechia
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