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
Phase:
Phase 2
Details
Lead Sponsor:
University of California, Los Angeles
Collaborators:
University of Rochester University of South Florida