Hope for rare disease: new drug shows promise in Long-Term study
NCT ID NCT06657859
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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.
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.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
Contacts and locations
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Locations
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Great Ormond Street Hospital
London, WC1N3JH, United Kingdom
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Hospital Sant Joan de Déu
Barcelona, Spain, 08950, Spain