Promising drug for rare balance disorder enters final testing phase

NCT ID NCT07185347

Recruiting now Disease control Sponsor: Assistance Publique - Hôpitaux de Paris Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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.

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.

Ataxia Diplopia spinocerebellar ataxia 27A spinocerebellar ataxia 27B, late-onset

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SPINOCEREBELLAR ATAXIA 27B (SCA27B)

Contacts and locations

Locations

Genetics Department, CHU de Bordeaux
RECRUITING

Bordeaux, France
Genetics Department, CHU de Rouen
NOT_YET_RECRUITING

Rouen, France
Genetics Department, Pitié-Salpêtrière University Hospital
RECRUITING

Paris, France
Neurology Department, CHRU de Strasbourg
NOT_YET_RECRUITING

Strasbourg, France
Neurology Department, CHU d'Angers
NOT_YET_RECRUITING

Angers, France
Neurology Department, CHU de Toulouse
NOT_YET_RECRUITING

Toulouse, France
Neurology Department, Gui De Chauliac Hospital
RECRUITING

Montpellier, France
Neurology Department, Hôpital Pierre Wertheimer Hospital
NOT_YET_RECRUITING

Lyon, France
Neurology and Gentics Department, CHU de Dijon
RECRUITING

Dijon, France