Trial Outcomes & Findings for Dalfampridine and Gait in Spinocerebellar Ataxias (NCT NCT01811706)
NCT ID: NCT01811706
Last Updated: 2015-01-12
Results Overview
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.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
20 participants
Primary outcome timeframe
Baseline and 4 weeks after Dalfampridine or placebo
Results posted on
2015-01-12
Participant Flow
Participant milestones
| Measure |
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.
|
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.
|
|---|---|---|
|
First Intervention ( 4 Weeks)
STARTED
|
10
|
10
|
|
First Intervention ( 4 Weeks)
COMPLETED
|
9
|
10
|
|
First Intervention ( 4 Weeks)
NOT COMPLETED
|
1
|
0
|
|
Washout ( 2 Weeks)
STARTED
|
9
|
10
|
|
Washout ( 2 Weeks)
COMPLETED
|
9
|
10
|
|
Washout ( 2 Weeks)
NOT COMPLETED
|
0
|
0
|
|
Second Intervention (4 Weeks)
STARTED
|
9
|
10
|
|
Second Intervention (4 Weeks)
COMPLETED
|
9
|
10
|
|
Second Intervention (4 Weeks)
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
| Measure |
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.
|
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.
|
|---|---|---|
|
First Intervention ( 4 Weeks)
Adverse Event
|
1
|
0
|
Baseline Characteristics
Dalfampridine and Gait in Spinocerebellar Ataxias
Baseline characteristics by cohort
| Measure |
All Study Participants
n=20 Participants
|
|---|---|
|
Age, Continuous
|
54.9 years
STANDARD_DEVIATION 14.3 • n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
20 participants
n=5 Participants
|
|
T25FW at screening visit
|
25.5 second
STANDARD_DEVIATION 9.7 • n=5 Participants
|
|
SARA at screening visit
|
11.1 point
STANDARD_DEVIATION 3.4 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline and 4 weeks after Dalfampridine or placeboPopulation: Patient with spinocerebellar ataxia (SCA) 1, 2, 3, or 6
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.
Outcome measures
| Measure |
Dalfampridine
n=19 Participants
Participant who received Dalfampridine at an oral dose of 10mg every 12 hours for 4 weeks period.
|
Placebo
n=19 Participants
Participant who received Placebo orally every 12 hours for a 4 week period.
|
|---|---|---|
|
Change in Timed 25 Feet Walking Test (T25FW)
T25FW at baseline
|
23.7 second
Standard Deviation 10.1
|
24.4 second
Standard Deviation 10.1
|
|
Change in Timed 25 Feet Walking Test (T25FW)
Change from baseline at 4 weeks
|
-1.1 second
Standard Deviation 4.7
|
-0.3 second
Standard Deviation 4.8
|
SECONDARY outcome
Timeframe: Baseline and 4 weeks after Dalfampridine or placeboScale 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.
Outcome measures
| Measure |
Dalfampridine
n=19 Participants
Participant who received Dalfampridine at an oral dose of 10mg every 12 hours for 4 weeks period.
|
Placebo
n=19 Participants
Participant who received Placebo orally every 12 hours for a 4 week period.
|
|---|---|---|
|
Change in Scale of Assessment and Rating of Ataxia (SARA)
SARA at baseline
|
10.0 point
Standard Deviation 3.5
|
10.1 point
Standard Deviation 3.3
|
|
Change in Scale of Assessment and Rating of Ataxia (SARA)
Change from baseline at week 4
|
0.6 point
Standard Deviation 1.5
|
0.8 point
Standard Deviation 1.8
|
SECONDARY outcome
Timeframe: Baseline and 4 weeks after Dalfampridine or placeboBiomechanical 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.
Outcome measures
| Measure |
Dalfampridine
n=19 Participants
Participant who received Dalfampridine at an oral dose of 10mg every 12 hours for 4 weeks period.
|
Placebo
n=19 Participants
Participant who received Placebo orally every 12 hours for a 4 week period.
|
|---|---|---|
|
Biomechanical Assessment of Gait (BAG)-Stride Length
Sstride Length at baseline
|
112.7 cm
Standard Deviation 29.4
|
111.8 cm
Standard Deviation 30.0
|
|
Biomechanical Assessment of Gait (BAG)-Stride Length
Stride Length at week 4
|
1.6 cm
Standard Deviation 9.4
|
4.0 cm
Standard Deviation 9.9
|
Adverse Events
Dalfampridine
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Dalfampridine
n=20 participants at risk
Participant received Dalfampridine at an oral dose of 10mg every 12 hours, for 4 weeks period.
|
Placebo
n=19 participants at risk
Participant received Placebo orally every 12 hours for a 4 week period.
|
|---|---|---|
|
Nervous system disorders
headache
|
15.0%
3/20 • Number of events 3 • 10 weeks per patient
|
5.3%
1/19 • Number of events 1 • 10 weeks per patient
|
|
Nervous system disorders
lethargy
|
5.0%
1/20 • Number of events 1 • 10 weeks per patient
|
0.00%
0/19 • 10 weeks per patient
|
|
Gastrointestinal disorders
dental caries
|
5.0%
1/20 • Number of events 1 • 10 weeks per patient
|
0.00%
0/19 • 10 weeks per patient
|
|
Musculoskeletal and connective tissue disorders
pain in extremity
|
5.0%
1/20 • Number of events 1 • 10 weeks per patient
|
0.00%
0/19 • 10 weeks per patient
|
|
Ear and labyrinth disorders
vertigo
|
0.00%
0/20 • 10 weeks per patient
|
5.3%
1/19 • Number of events 1 • 10 weeks per patient
|
|
Nervous system disorders
tremor
|
0.00%
0/20 • 10 weeks per patient
|
5.3%
1/19 • Number of events 1 • 10 weeks per patient
|
|
Gastrointestinal disorders
constipation
|
0.00%
0/20 • 10 weeks per patient
|
5.3%
1/19 • Number of events 1 • 10 weeks per patient
|
|
Gastrointestinal disorders
dry mouth
|
0.00%
0/20 • 10 weeks per patient
|
5.3%
1/19 • Number of events 1 • 10 weeks per patient
|
|
General disorders
gait disturbance
|
5.0%
1/20 • Number of events 1 • 10 weeks per patient
|
21.1%
4/19 • Number of events 4 • 10 weeks per patient
|
|
Musculoskeletal and connective tissue disorders
arthralgia
|
5.0%
1/20 • Number of events 1 • 10 weeks per patient
|
0.00%
0/19 • 10 weeks per patient
|
|
Skin and subcutaneous tissue disorders
hyperhidrosis
|
5.0%
1/20 • Number of events 1 • 10 weeks per patient
|
0.00%
0/19 • 10 weeks per patient
|
|
General disorders
chills
|
0.00%
0/20 • 10 weeks per patient
|
5.3%
1/19 • Number of events 1 • 10 weeks per patient
|
|
Nervous system disorders
somnolence
|
0.00%
0/20 • 10 weeks per patient
|
5.3%
1/19 • Number of events 1 • 10 weeks per patient
|
|
Renal and urinary disorders
urinary frequency
|
0.00%
0/20 • 10 weeks per patient
|
5.3%
1/19 • Number of events 1 • 10 weeks per patient
|
|
Eye disorders
blurred vision
|
5.0%
1/20 • Number of events 1 • 10 weeks per patient
|
0.00%
0/19 • 10 weeks per patient
|
|
Respiratory, thoracic and mediastinal disorders
voice alteration
|
5.0%
1/20 • Number of events 1 • 10 weeks per patient
|
0.00%
0/19 • 10 weeks per patient
|
|
Gastrointestinal disorders
vomiting
|
0.00%
0/20 • 10 weeks per patient
|
5.3%
1/19 • Number of events 1 • 10 weeks per patient
|
Additional Information
Dr. Guangbin Xia, Assistant Professor
University of Florida
Phone: 352-273-5550
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place