Dalfampridine and Gait in Spinocerebellar Ataxias

Therapeutic Effect of Dalfampridine on Gait Incoordination in Spinocerebellar Ataxias- A Randomized, Double-blinded, Placebo-controlled, Crossover Clinical Trial

Investigators expect there will be improvement in walking speed and steadiness after taking Dalfampridine, thereby improving activities of daily living and enhancing social and occupational functions for patients with spinocerebellar ataxia.

Study Overview

• Status: Completed
• Conditions: Spinocerebellar Ataxias Type 1; Spinocerebellar Ataxias Type 2; Spinocerebellar Ataxias Type 3; Spinocerebellar Ataxias Type 6
• Intervention / Treatment: Drug: Dalfampridine; Drug: Placebo

Detailed Description

Twenty spinocerebellar ataxia patients will be randomized to receive either Dalfampridine or placebo over a total period of 10 weeks. After entering the study, patients will return every 2 weeks for evaluation. After four weeks, intervention will be stopped and patient will enter a 2-week wash out period where they do not take any drug. Then, patients will be given the opposite treatment (Dalfampridine or placebo) and this "crossover" study will be performed for another 4 weeks. Investigators expect there will be improvement in walking speed and steadiness after taking Dalfampridine, thereby improving activities of daily living and enhancing social and occupational functions.

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Florida
    • Gainesville, Florida, United States, 32607
      • University of Florida

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Individuals at age 18 years or older.
• Individuals who can provide the informed consent
• Genetic confirmed definite spinocerebellar ataxias (SCA)
• Able to complete two trials of the timed 25-foot walk at screening

Exclusion Criteria:

• Patients who has severe ataxia and unable to ambulate.
• Any orthopedic condition that would affect motor performance.
• Patients with secondary ataxia from general medical disorders
• Individuals who have major psychiatric disorders that prevents compliance
• History of epilepsy
• Patients with active drug or alcohol use or dependence that would interfere with adherence to study requirements
• Inability or unwillingness of subject or legal guardian/representative to give written informed consent.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Dalfampridine and then placebo; Participant first receive Dalfampridine at an oral dose of 10mg every 12 hours, for 4 weeks. After a washout period of 2 weeks, they then receive Placebo tablet orally every 12 hours, for a 4 weeks period.	Drug: Dalfampridine; Dalfampridine will be provided at an oral dose of 10mg every 12 hours, for 4 weeks period; Other Names: Ampyra; Drug: Placebo; Placebo will be administered orally every 12 hours, for a 4 week period.; Other Names: Sugar Pill
Experimental: Placebo, Then Dalfampridine; Participant first receive Placebo tablet orally every 12 hours, for a 4 weeks period. After a washout period of 2 weeks, they then receive Dalfampridine at an oral dose of 10mg every 12 hours, for 4 weeks.	Drug: Dalfampridine; Dalfampridine will be provided at an oral dose of 10mg every 12 hours, for 4 weeks period; Other Names: Ampyra; Drug: Placebo; Placebo will be administered orally every 12 hours, for a 4 week period.; Other Names: Sugar Pill

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Timed 25 Feet Walking Test (T25FW)	The patient is directed to one end of a clearly marked 25-foot course and is instructed to walk 25 feet as quickly as possible, but safely. The time, in seconds, is calculated from the initiation of the instruction to start and ends when the patient has reached the 25-foot mark. Baseline values are recorded twice. One was at the beginning of the intervention. The second was 2 weeks after washout period and before the second intervention.	Baseline and 4 weeks after Dalfampridine or placebo

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Scale of Assessment and Rating of Ataxia (SARA)	Scale for the assessment and rating of ataxia (SARA) is a clinical scale that is based on a semiquantitative assessment of cerebellar ataxia on an impairment level. SARA has 8 items that are related to gait, stance, sitting, speech, finger-chase test, nose-finger test, fast alternating movements and heel-shin test. SARA score ranges from 0 to 40, with higher scores indicating more severe disease.	Baseline and 4 weeks after Dalfampridine or placebo
Biomechanical Assessment of Gait (BAG)-Stride Length	Biomechanical Assessment of Gait is a sensitive, quantitative movement analysis system. Stride length was analyzed. Baseline values are recorded twice. One was at the beginning of the intervention. The second was 2 weeks after washout period and before the second intervention.	Baseline and 4 weeks after Dalfampridine or placebo

Collaborators and Investigators

• Sponsor: University of Florida
• Collaborators: Acorda Therapeutics
• Investigators: Principal Investigator: Guangbin Xia, MD, PhD, University of Florida

Study record dates

Study Major Dates

• Study Start: February 1, 2013
• Primary Completion (Actual): December 1, 2013
• Study Completion (Actual): December 1, 2013

Study Registration Dates

• First Submitted: March 6, 2013
• First Submitted That Met QC Criteria: March 13, 2013
• First Posted (Estimate): March 14, 2013

Study Record Updates

• Last Update Posted (Estimate): January 12, 2015
• Last Update Submitted That Met QC Criteria: January 8, 2015
• Last Verified: January 1, 2015

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Genetic Diseases, Inborn; Neurodegenerative Diseases; Dyskinesias; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Cerebellar Diseases; Ataxia; Cerebellar Ataxia; Spinocerebellar Ataxias; Spinocerebellar Degenerations; Machado-Joseph Disease; Molecular Mechanisms of Pharmacological Action; Membrane Transport Modulators; Potassium Channel Blockers; 4-Aminopyridine
• Other Study ID Numbers: 20121107; 1133511 (Other Identifier: WIRB Study Number)