Can a new infusion ease muscle weakness in rare genetic disorders?
NCT ID NCT07746089
First seen Aug 04, 2026 · Last updated Aug 05, 2026 · Updated 1 time
Summary
This Phase 3 trial is testing whether an experimental drug called adimanebart can help people aged 12 and older with certain congenital myasthenic syndromes (CMS) feel better and do daily activities more easily. Participants are randomly assigned to receive either adimanebart or a placebo, and after that, everyone can receive the drug in an open-label phase. The study measures changes in walking distance and physical function over about 24 weeks.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Adimanebart given as an intravenous infusion
- What this could lead to
- If it works, adimanebart could become a new treatment to ease muscle weakness and improve daily activities for people with certain congenital myasthenic syndromes.
- What could go wrong
- This is an early-stage test in a small group, and results may not hold up. The drug could also cause side effects, and not everyone may respond.
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
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Study contacts
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Contact
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