4-Aminopyridine in Episodic Ataxia Type 2

NCT01543750 | Withdrawn Phase 2 DMC

• 2 other identifiers: CINCH-EA2R01FD003923
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 3

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

Conditions

Episodic Ataxia Type 2

Keywords

episodic ataxia; CACNA1A mutations

Outcome Measures

Primary Outcomes (1)

• the frequency of ataxia episodes

  Trial participants have frequent episodes of ataxia at baseline. The participants will document daily whether ataxia events occurred during the 2-month screening period and the 9-month study period by calling a toll-free number and participating in an Interactive Voice Response (IVR) system.

  11 months

Secondary Outcomes (6)

• impact on daily activities

  11 months

• duration of ataxia episodes

  11 months

• severity of ataxia episodes

  11 months

• treatment satisfaction

  9 months

• Toxicity

  9 months

Study Arms (2)

Study Medication

EXPERIMENTAL

4-aminopyridine 10mg twice daily for 8 weeks

Drug: 4-Aminopyridine

Placebo

EXPERIMENTAL

placebo twice daily for 8 weeks

Drug: Placebo

Interventions

4-Aminopyridine DRUG

Study Medication

Placebo DRUG

Placebo

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients will be included if they:
• Have EA2 genetically confirmed to harbor mutations in CACNA1A
• Are ≥ 18 years of age
• Are not taking acetazolamide (because of intolerance, poor response, or allergy)
• Are able to maintain a daily log of ataxia episode(s) and report daily by using an Interactive Voice Recording System (IVR) throughout the study
• Experience ≥ 3 ataxia episodes per month during the two-month screening period to qualify for randomization

You may not qualify if:

• Patients will be excluded if they:
• Have seizures or a history of seizures
• Have first-degree relatives with EA2 and seizures
• Have renal disease with impaired function (Creatinine clearance CrCl≤50ml/min)
• Are pregnant or breast feeding (women of childbearing age will be tested for pregnancy and must be using birth control)
• Are unable to comply with the study requirement

Sponsors & Collaborators

• University of California, Los Angeles: lead
• University of Rochester: collaborator
• University of South Florida: collaborator

Study Sites (3)

University of California, Los Angeles (UCLA)

Los Angeles, California, 90095, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

University of Rochester School of Medicine

Rochester, New York, 14642, United States

Location

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MeSH Terms

Conditions

Episodic Ataxia, Type 2; Episodic Ataxia

Interventions

4-Aminopyridine

Intervention Hierarchy (Ancestors)

Aminopyridines; Amines; Organic Chemicals; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Joanna C Jen, MD PhD

  University of California, Los Angeles

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 27, 2011
• First Posted: March 5, 2012
• Last Updated: November 16, 2020

Record last verified: 2020-11

Locations

US (3)