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Dalfampridine and Gait in Spinocerebellar Ataxias

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ClinicalTrials.gov Identifier: NCT01811706
Recruitment Status : Completed
First Posted : March 14, 2013
Results First Posted : January 12, 2015
Last Update Posted : January 12, 2015
Sponsor:
Collaborator:
Acorda Therapeutics
Information provided by (Responsible Party):
University of Florida

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Spinocerebellar Ataxias Type 1
Spinocerebellar Ataxias Type 2
Spinocerebellar Ataxias Type 3
Spinocerebellar Ataxias Type 6
Interventions Drug: Dalfampridine
Drug: Placebo
Enrollment 20
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Dalfampridine and Then Placebo Placebo, Then Dalfampridine
Hide Arm/Group Description

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.

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.

Period Title: First Intervention ( 4 Weeks)
Started 10 10
Completed 9 10
Not Completed 1 0
Reason Not Completed
Adverse Event             1             0
Period Title: Washout ( 2 Weeks)
Started 9 10
Completed 9 10
Not Completed 0 0
Period Title: Second Intervention (4 Weeks)
Started 9 10
Completed 9 10
Not Completed 0 0
Arm/Group Title All Study Participants
Hide Arm/Group Description [Not Specified]
Overall Number of Baseline Participants 20
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 20 participants
54.9  (14.3)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
Female
8
  40.0%
Male
12
  60.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 20 participants
20
T25FW at screening visit   [1] 
Mean (Standard Deviation)
Unit of measure:  Second
Number Analyzed 20 participants
25.5  (9.7)
[1]
Measure Description: 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.
SARA at screening visit   [1] 
Mean (Standard Deviation)
Unit of measure:  Point
Number Analyzed 20 participants
11.1  (3.4)
[1]
Measure Description: 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.
1.Primary Outcome
Title Change in Timed 25 Feet Walking Test (T25FW)
Hide Description 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.
Time Frame Baseline and 4 weeks after Dalfampridine or placebo
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patient with spinocerebellar ataxia (SCA) 1, 2, 3, or 6
Arm/Group Title Dalfampridine Placebo
Hide Arm/Group Description:
Participant who received Dalfampridine at an oral dose of 10mg every 12 hours for 4 weeks period.
Participant who received Placebo orally every 12 hours for a 4 week period.
Overall Number of Participants Analyzed 19 19
Mean (Standard Deviation)
Unit of Measure: second
T25FW at baseline 23.7  (10.1) 24.4  (10.1)
Change from baseline at 4 weeks -1.1  (4.7) -0.3  (4.8)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Dalfampridine, Placebo
Comments Null hypothesis is that there was no difference in change of T25FW between Dalfampridine and Placebo. The test was performed with a significant level of 0.05 (two-sided).
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value >0.05
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
2.Secondary Outcome
Title Change in Scale of Assessment and Rating of Ataxia (SARA)
Hide Description 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.
Time Frame Baseline and 4 weeks after Dalfampridine or placebo
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Dalfampridine Placebo
Hide Arm/Group Description:
Participant received Dalfampridine at an oral dose of 10mg every 12 hours, for 4 weeks period.
Participant received Placebo orally every 12 hours for a 4 week period.
Overall Number of Participants Analyzed 19 19
Mean (Standard Deviation)
Unit of Measure: point
SARA at baseline 10.0  (3.5) 10.1  (3.3)
Change from baseline at week 4 0.6  (1.5) 0.8  (1.8)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Dalfampridine, Placebo
Comments Null hypothesis is that there was no difference in change of SARA score between Dalfampridine and Placebo. The test was performed with a significant level of 0.05 (two-sided).
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value >0.05
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
3.Secondary Outcome
Title Biomechanical Assessment of Gait (BAG)-Stride Length
Hide Description 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.
Time Frame Baseline and 4 weeks after Dalfampridine or placebo
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Dalfampridine Placebo
Hide Arm/Group Description:
Participant who received Dalfampridine at an oral dose of 10mg every 12 hours for 4 weeks period.
Participant who received Placebo orally every 12 hours for a 4 week period.
Overall Number of Participants Analyzed 19 19
Mean (Standard Deviation)
Unit of Measure: cm
Sstride Length at baseline 112.7  (29.4) 111.8  (30.0)
Stride Length at week 4 1.6  (9.4) 4.0  (9.9)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Dalfampridine, Placebo
Comments Null hypothesis is that there was no difference in change of Stride Length on BAG between Dalfampridine and Placebo. The test was performed with a significant level of 0.05 (two-sided).
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value >0.05
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Time Frame 10 weeks per patient
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Dalfampridine Placebo
Hide Arm/Group Description Participant received Dalfampridine at an oral dose of 10mg every 12 hours, for 4 weeks period. Participant received Placebo orally every 12 hours for a 4 week period.
All-Cause Mortality
Dalfampridine Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Dalfampridine Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/20 (0.00%)      0/19 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Dalfampridine Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/20 (35.00%)      8/19 (42.11%)    
Ear and labyrinth disorders     
vertigo * 1 [1]  0/20 (0.00%)  0 1/19 (5.26%)  1
Eye disorders     
blurred vision * 1 [2]  1/20 (5.00%)  1 0/19 (0.00%)  0
Gastrointestinal disorders     
dental caries * 1 [3]  1/20 (5.00%)  1 0/19 (0.00%)  0
constipation * 1 [4]  0/20 (0.00%)  0 1/19 (5.26%)  1
dry mouth * 1 [4]  0/20 (0.00%)  0 1/19 (5.26%)  1
vomiting * 1 [1]  0/20 (0.00%)  0 1/19 (5.26%)  1
General disorders     
gait disturbance * 1 [5]  1/20 (5.00%)  1 4/19 (21.05%)  4
chills * 1 [1]  0/20 (0.00%)  0 1/19 (5.26%)  1
Musculoskeletal and connective tissue disorders     
pain in extremity * 1 [6]  1/20 (5.00%)  1 0/19 (0.00%)  0
arthralgia * 1 [7]  1/20 (5.00%)  1 0/19 (0.00%)  0
Nervous system disorders     
headache * 1 [8]  3/20 (15.00%)  3 1/19 (5.26%)  1
lethargy * 1 [1]  1/20 (5.00%)  1 0/19 (0.00%)  0
tremor * 1 [9]  0/20 (0.00%)  0 1/19 (5.26%)  1
somnolence * 1 [1]  0/20 (0.00%)  0 1/19 (5.26%)  1
Renal and urinary disorders     
urinary frequency * 1 [1]  0/20 (0.00%)  0 1/19 (5.26%)  1
Respiratory, thoracic and mediastinal disorders     
voice alteration * 1 [10]  1/20 (5.00%)  1 0/19 (0.00%)  0
Skin and subcutaneous tissue disorders     
hyperhidrosis * 1 [1]  1/20 (5.00%)  1 0/19 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (4.0)
[1]
grade 1
[2]
grade 1 accommodation issues
[3]
grade 3 - tooth removal
[4]
grade 2
[5]
all grade 1
[6]
increased pain in the left leg
[7]
grade 1 - knee pain
[8]
Two grade 1 headaches, and one grade 2 headache.
[9]
Grade 1 tremor of finger
[10]
grade 2 - slurring of speech
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. Guangbin Xia, Assistant Professor
Organization: University of Florida
Phone: 352-273-5550
Responsible Party: University of Florida
ClinicalTrials.gov Identifier: NCT01811706     History of Changes
Other Study ID Numbers: 20121107
1133511 ( Other Identifier: WIRB Study Number )
First Submitted: March 6, 2013
First Posted: March 14, 2013
Results First Submitted: December 10, 2014
Results First Posted: January 12, 2015
Last Update Posted: January 12, 2015