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| ID | Type | Description | Link |
|---|---|---|---|
| 1133511 | Other Identifier | WIRB Study Number |
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| Name | Class |
|---|---|
| Acorda Therapeutics | INDUSTRY |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dalfampridine and then placebo | Experimental | 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. |
|
| Placebo, Then Dalfampridine | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Dalfampridine | Drug | Dalfampridine will be provided at an oral dose of 10mg every 12 hours, for 4 weeks period |
|
| 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 |
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Guangbin Xia, MD, PhD | University of Florida | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Florida | Gainesville | Florida | 32607 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. Dalfampridine: Dalfampridine will be provided at an oral dose of 10mg every 12 hours, for 4 weeks period Placebo: Placebo will be administered orally every 12 hours, for a 4 week period. |
| FG001 | 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. Dalfampridine: Dalfampridine will be provided at an oral dose of 10mg every 12 hours, for 4 weeks period Placebo: Placebo will be administered orally every 12 hours, for a 4 week period. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| First Intervention ( 4 Weeks) |
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| Washout ( 2 Weeks) |
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| Second Intervention (4 Weeks) |
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| ID | Title | Description |
|---|---|---|
| BG000 | All Study Participants |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Patient with spinocerebellar ataxia (SCA) 1, 2, 3, or 6 | Posted | Mean | Standard Deviation | second | Baseline and 4 weeks after Dalfampridine or placebo |
|
10 weeks per patient
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Dalfampridine | Participant received Dalfampridine at an oral dose of 10mg every 12 hours, for 4 weeks period. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| headache | Nervous system disorders | CTCAE (4.0) | Non-systematic Assessment | Two grade 1 headaches, and one grade 2 headache. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Guangbin Xia, Assistant Professor | University of Florida | 352-273-5550 | guangbin.xia@neurology.ufl.edu |
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| ID | Term |
|---|---|
| D015761 | 4-Aminopyridine |
| D000073893 | Sugars |
| ID | Term |
|---|---|
| D000631 | Aminopyridines |
| D000588 | Amines |
| D009930 | Organic Chemicals |
| D011725 | Pyridines |
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| Placebo | Drug | Placebo will be administered orally every 12 hours, for a 4 week period. |
|
|
| 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 |
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| years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| T25FW at screening visit | Timed 25-Foot Walk (T25FW): The patient is directed to one end of a 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. | Mean | Standard Deviation | second |
|
| SARA at screening visit | 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. | Mean | Standard Deviation | point |
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| Placebo |
Participant who received Placebo orally every 12 hours for a 4 week period. |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | point | Baseline and 4 weeks after Dalfampridine or placebo |
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|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | cm | Baseline and 4 weeks after Dalfampridine or placebo |
|
|
|
|
| 0 |
| 20 |
| 7 |
| 20 |
| EG001 | Placebo | Participant received Placebo orally every 12 hours for a 4 week period. | 0 | 19 | 8 | 19 |
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| lethargy | Nervous system disorders | CTCAE (4.0) | Non-systematic Assessment | grade 1 |
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| dental caries | Gastrointestinal disorders | CTCAE (4.0) | Non-systematic Assessment | grade 3 - tooth removal |
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| pain in extremity | Musculoskeletal and connective tissue disorders | CTCAE (4.0) | Non-systematic Assessment | increased pain in the left leg |
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| vertigo | Ear and labyrinth disorders | CTCAE (4.0) | Non-systematic Assessment | grade 1 |
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| tremor | Nervous system disorders | CTCAE (4.0) | Non-systematic Assessment | Grade 1 tremor of finger |
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| constipation | Gastrointestinal disorders | CTCAE (4.0) | Non-systematic Assessment | grade 2 |
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| dry mouth | Gastrointestinal disorders | CTCAE (4.0) | Non-systematic Assessment | grade 2 |
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| gait disturbance | General disorders | CTCAE (4.0) | Non-systematic Assessment | all grade 1 |
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| arthralgia | Musculoskeletal and connective tissue disorders | CTCAE (4.0) | Non-systematic Assessment | grade 1 - knee pain |
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| hyperhidrosis | Skin and subcutaneous tissue disorders | CTCAE (4.0) | Non-systematic Assessment | grade 1 |
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| chills | General disorders | CTCAE (4.0) | Non-systematic Assessment | grade 1 |
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| somnolence | Nervous system disorders | CTCAE (4.0) | Non-systematic Assessment | grade 1 |
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| urinary frequency | Renal and urinary disorders | CTCAE (4.0) | Non-systematic Assessment | grade 1 |
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| blurred vision | Eye disorders | CTCAE (4.0) | Non-systematic Assessment | grade 1 accommodation issues |
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| voice alteration | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Non-systematic Assessment | grade 2 - slurring of speech |
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| vomiting | Gastrointestinal disorders | CTCAE (4.0) | Non-systematic Assessment | grade 1 |
|
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| D006573 |
| Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D002241 | Carbohydrates |