Promising drug for rare balance disorder enters final testing phase
NCT ID NCT07185347
First seen Nov 01, 2025 · Last updated Jun 22, 2026 · Updated 33 times
Summary
This Phase 3 trial tests whether fampridine can improve movement and vision in people with spinocerebellar ataxia SCA27B, a rare genetic condition that affects balance and coordination. About 70 adults will take either fampridine or a placebo twice daily for 12 weeks. The main goal is to see if more people on the drug show meaningful improvement on a standard ataxia rating scale.
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This is a summary of
the original study
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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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Contacts and locations
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Study contacts
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Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Genetics Department, CHU de Bordeaux
RECRUITINGBordeaux, France
Contact
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Genetics Department, CHU de Rouen
NOT_YET_RECRUITINGRouen, France
Contact
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Genetics Department, Pitié-Salpêtrière University Hospital
RECRUITINGParis, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Neurology Department, CHRU de Strasbourg
NOT_YET_RECRUITINGStrasbourg, France
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Neurology Department, CHU d'Angers
NOT_YET_RECRUITINGAngers, France
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Neurology Department, CHU de Toulouse
NOT_YET_RECRUITINGToulouse, France
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Neurology Department, Gui De Chauliac Hospital
RECRUITINGMontpellier, France
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Neurology Department, Hôpital Pierre Wertheimer Hospital
NOT_YET_RECRUITINGLyon, France
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Contact
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Neurology and Gentics Department, CHU de Dijon
RECRUITINGDijon, France
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What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Fampridine (a potassium channel blocker, also known as 4-aminopyridine)
What this could lead to
If it works, this could provide a treatment to improve walking and reduce vision problems in people with SCA27B ataxia.
What could go wrong
This is a Phase 3 trial, but earlier positive results were from small, open-label studies. The drug may not work better than placebo, and side effects like hypersensitivity are possible.
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.