Dalfampridine and Gait in Spinocerebellar Ataxias
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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 |
- 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.
- 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.
- 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.
| Estimated Enrollment: | 30 |
| Study Start Date: | February 2013 |
| Estimated Study Completion Date: | October 2013 |
| Estimated Primary Completion Date: | October 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 |
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| Contact: Camille Swartz, BA | 352-273-5612 | cami.swartz@neurology.ufl.edu |
| Contact: Kyle Rizer, BA | 352-294-5194 | kyle.rizer@neurology.ufl.edu |
| United States, Florida | |
| University of Florida | Recruiting |
| Gainesville, Florida, United States, 32607 | |
| Contact: Camille Swartz, BA 352-273-5612 cami.swartz@neurology.ufl.edu | |
| Contact: Kyle Rizer, BA 352-294-5194 kyle.rizer@neurology.ufl.edu | |
| Principal Investigator: Guangbin Xia, MD, PhD | |
| 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: | March 13, 2013 |
| Health Authority: | United States: Food and Drug Administration United States: Institutional Review Board |
Additional relevant MeSH terms:
|
Ataxia Machado-Joseph Disease Spinocerebellar Ataxias Spinocerebellar Degenerations Dyskinesias Neurologic Manifestations Nervous System Diseases Signs and Symptoms Cerebellar Ataxia Cerebellar Diseases Brain Diseases Central Nervous System Diseases |
Spinal Cord Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn 4-Aminopyridine Potassium Channel Blockers Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action Pharmacologic Actions Cardiovascular Agents Therapeutic Uses |
ClinicalTrials.gov processed this record on June 18, 2013