Dalfampridine and Gait in Spinocerebellar Ataxias

This study has been completed.
Sponsor:
Collaborator:
Acorda Therapeutics
Information provided by (Responsible Party):
University of Florida
ClinicalTrials.gov Identifier:
NCT01811706
First received: March 6, 2013
Last updated: July 2, 2014
Last verified: July 2014
  Purpose

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.


Condition Intervention
Spinocerebellar Ataxias Type 1
Spinocerebellar Ataxias Type 2
Spinocerebellar Ataxias Type 3
Spinocerebellar Ataxias Type 6
Drug: Dalfampridine
Drug: Placebo

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Therapeutic Effect of Dalfampridine on Gait Incoordination in Spinocerebellar Ataxias- A Randomized, Double-blinded, Placebo-controlled, Crossover Clinical Trial

Resource links provided by NLM:


Further study details as provided by University of Florida:

Primary Outcome Measures:
  • Change in Timed 25 Feet Walking Test (T25FW) [ Time Frame: Screening and week 10 ] [ Designated as safety issue: No ]
    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 is calculated from the initiation of the instruction to start and ends when the patient has reached the 25-foot mark. Patients may use walking assistance devices if needed.

  • Change in Biomechanical Assessment of Gait (BAG) [ Time Frame: Screening and week 10 ] [ Designated as safety issue: No ]
    Biomechanical Assessment of Gait is a sensitive, quantitative movement analysis system. The analysis includes walking velocity, stride length, step width, double support and single support times, and center of mass displacement.

  • Change in Balance Test (BT) [ Time Frame: Screening and week 10 ] [ Designated as safety issue: No ]
    For 20 seconds, patients will stand on a pad that measures sway and tests balance, once with eyes open and once with eyes closed.


Secondary Outcome Measures:
  • Change in Scale of Assessment and Rating of Ataxia (SARA) [ Time Frame: Screening and week 10 ] [ Designated as safety issue: No ]
    SARA is a clinical scale including 8 items that are related to gait, stance, sitting, speech, finger-chase test, nose-finger test, fast alternating movements and heel-shin test.

  • Change in Karnofsky Performance Scale (KPS) [ Time Frame: Screening and week 10 ] [ Designated as safety issue: No ]
    Karnofsky Performance Scale (KPS) will be used to measure functional status. It was designed to measure the level of patient activity and medical care requirements. It is a general measure of patient independence.

  • Change in World Health Organization Quality of Life (WHOQOL) Questionnaire [ Time Frame: Screening and week 10 ] [ Designated as safety issue: No ]
    The WHOQOL questionnaire is an international cross-culturally comparable quality of life assessment instrument. It assesses the individual's perceptions in the context of their culture and value systems, and their personal goals, standards and concerns.


Other Outcome Measures:
  • Dalfampridine plasma level [ Time Frame: week 4 and week 10 ] [ Designated as safety issue: No ]
    This is a blood test to monitor compliance by looking at Dalfampridine plasma level.

  • Adverse Events [ Time Frame: Weeks 2, 4, 6, 8, 10 ] [ Designated as safety issue: Yes ]
    Patients will report on any adverse events they experienced that may or may not be due to treatment so that we may monitor safety.


Enrollment: 20
Study Start Date: February 2013
Study Completion Date: December 2013
Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Dalfampridine
Dalfampridine will be provided 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.
Other Name: Dalfampridine
Placebo Comparator: Placebo
Placebo will be administered orally every 12 hours, for a 4 week period.
Drug: Placebo
Placebo will be administered orally every 12 hours, for a 4 week period.

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 five 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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01811706

Locations
United States, Florida
University of Florida
Gainesville, Florida, United States, 32607
Sponsors and Collaborators
University of Florida
Acorda Therapeutics
Investigators
Principal Investigator: Guangbin Xia, MD, PhD University of Florida
  More Information

No publications provided

Responsible Party: University of Florida
ClinicalTrials.gov Identifier: NCT01811706     History of Changes
Other Study ID Numbers: 20121107, 1133511
Study First Received: March 6, 2013
Last Updated: July 2, 2014
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

Additional relevant MeSH terms:
Ataxia
Spinocerebellar Ataxias
Cerebellar Ataxia
Machado-Joseph Disease
Spinocerebellar Degenerations
Brain Diseases
Central Nervous System Diseases
Cerebellar Diseases
Dyskinesias
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Nervous System Diseases
Neurodegenerative Diseases
Neurologic Manifestations
Signs and Symptoms
Spinal Cord Diseases
4-Aminopyridine
Cardiovascular Agents
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Potassium Channel Blockers
Therapeutic Uses

ClinicalTrials.gov processed this record on October 29, 2014