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Withdrawn Phase 2 Interventional

4-Aminopyridine in Episodic Ataxia Type 2

ClinicalTrials.gov ID: NCT01543750

Public ClinicalTrials.gov record NCT01543750. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 7, 2026, 3:44 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

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

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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

Study identification

NCT ID
NCT01543750
Recruitment status
Withdrawn
Study type
Interventional
Phase
Phase 2
Enrollment
Not listed

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
18 Years and older
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Not listed
Primary completion
Not listed
Completion
Not listed
Last update posted
Nov 15, 2020

United States locations

U.S. sites
3
U.S. states
3
U.S. cities
3
Facility City State ZIP Site status
University of California, Los Angeles (UCLA) Los Angeles California 90095
University of South Florida Tampa Florida 33612
University of Rochester School of Medicine Rochester New York 14642

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT01543750, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Nov 15, 2020 · Synced Sep 7, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT01543750 live on ClinicalTrials.gov.

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