Clinical Trial: 4-Aminopyridine in Episodic Ataxia Type 2

Study Status: Completed
Recruit Status: Unknown status
Study Type: Interventional




Official Title: Phase 2 Study of 4-Aminopyridine for the Treatment of Episodic Ataxia Type 2

Brief Summary: Episodic ataxia type 2 (EA2) is a rare familial neurological condition characterized by debilitating episodes of vertigo and imbalance. Since the serendipitous discovery of dramatic response of EA2 to acetazolamide, acetazolamide has been the first-line treatment for EA2. Yet, for those patients who do not respond to or cannot tolerate acetazolamide, there is no alternative treatment. The purpose of this randomized trial is to test whether 4-aminopyridine may reduce the ataxia episodes in EA2 as an alternative to acetazolamide. Funding Source - FDA OOPD