Effect of Dalfampridine in Patients With Hereditary Spastic Paraplegia
Primary Purpose
Hereditary Spastic Paraplegia
Status
Completed
Phase
Not Applicable
Locations
Cyprus
Study Type
Interventional
Intervention
Dalfampridine 10 MG
Placebo
Physiotherapy
Sponsored by
About this trial
This is an interventional treatment trial for Hereditary Spastic Paraplegia
Eligibility Criteria
Inclusion Criteria: Diagnosis of Hereditary Spastic Paraplegia at least 1 year ago Exclusion Criteria: Having another neurological disorder An orthopedic deformity in the lower extremity Having a serious cognitive impairment
Sites / Locations
- Dr. Burhan Nalbantoğlu State Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Medication
No Medication
Arm Description
Dalfampridine plus physiotherapy
Placebo plus physiotherapy
Outcomes
Primary Outcome Measures
Timed 25-foot Walk Test
The Timed 25-foot walk test (T25FW) is considered the "best characterized objective measure of walking disability and can be used across a wide range of walking disabilities". For the T25-FW, patients were instructed to walk as fast as they could in a safemanner along amarked 25-foot course. The time in seconds to complete each test was recorded, and the test was immediately repeated.
Secondary Outcome Measures
Sit to Stand Test
30 second Chair-Stand Test which is a reliable and valid measure used to assess lower extremity strength and endurance is used where the number of sitting and getting up within 30 seconds gives the score of the test.
Timed Up and Go test
Functional mobility was evaluated using the Timed up and Go Test 'TUG' test, which is also reliable and valid test for people with Parkinson's disease. (Morris2001; Van2016). Upon issuing the command "Go," the participants stood up from a normal chair, walked 3 meters, turned, walked back to the chair, and sat. The time began with the command "Go" and ended when the participants sat back to the chair. This test was repeated three times, and the shortest performance time was recorded
Modified Ashworth Scale
Modified Ashworth Scale (MAS) is one of the reliable and valid methods to measure muscle spasticity. The procedure to evaluate specific muscle groups; passively moved through the range of motion of a limb, and the resistance encountered during muscle stretch is rated on a five-point scale. Ashworth defines this rating as; 0 = no increase in tone, 1 = slight increase in tone at the end of the range of motion, 1+ = slight increase in tone throughout less than half the range of motion, 2 = increased muscle tone throughout the full range of motion, but passive movement is present. 3=tone movement that makes passive movement difficult, 4=rigidity .
Muscle Length Measurement
Bilateral muscle length measurements were obtained using a standard goniometer. The patient positioned in supine position; the dorsiflexion has been tested for the gastrocnemius, the straight leg rise - hip angle has been measured to assess hamstrings, the Thomas test was used to assess iliopsoas and hip abduction angle was used for adductor group muscles.
Full Information
NCT ID
NCT05613114
First Posted
November 4, 2022
Last Updated
November 17, 2022
Sponsor
European University of Lefke
1. Study Identification
Unique Protocol Identification Number
NCT05613114
Brief Title
Effect of Dalfampridine in Patients With Hereditary Spastic Paraplegia
Official Title
Effect of Dalfampridine in Patients With Hereditary Spastic Paraplegia
Study Type
Interventional
2. Study Status
Record Verification Date
November 2022
Overall Recruitment Status
Completed
Study Start Date
August 3, 2020 (Actual)
Primary Completion Date
March 12, 2021 (Actual)
Study Completion Date
March 12, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
European University of Lefke
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
There are limited but encouraging results supporting the use of dalfampridine in patients with hereditary spastic paraplegia. The investigators aimed to investigate the effects of dalfampridine on walking speed, muscle length, spasticity, functional strength, and functional mobility in patients with hereditary spastic paraplegia. In this triple-blinded, randomized, placebo-controlled trial, 4 patients with hereditary spastic paraplegia received dalfampridine (10 mg twice daily) plus physiotherapy (2 times per week), and 4 patients received placebo plus physiotherapy for a total duration of 8 weeks. The assessor and treating physiotherapists, and patients were masked to the group allocation. The primary outcome was Timed 25-foot Walk Test at the end of the 8-week treatment. The secondary outcome measures were functional mobility, functional muscle strength, muscle length, and spasticity.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Spastic Paraplegia
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderOutcomes Assessor
Allocation
Randomized
Enrollment
8 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Medication
Arm Type
Experimental
Arm Description
Dalfampridine plus physiotherapy
Arm Title
No Medication
Arm Type
Placebo Comparator
Arm Description
Placebo plus physiotherapy
Intervention Type
Drug
Intervention Name(s)
Dalfampridine 10 MG
Intervention Description
The participants in the experimental group received dalfampridine administered as 10 mg extended-release tablets every twelve hours for 8 weeks.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Control group received a placebo drug with the same administration method (2 times per week for the total duration of 8 weeks).
Intervention Type
Behavioral
Intervention Name(s)
Physiotherapy
Intervention Description
Conventional physiotherapy program including stretching and flexibility, strengthening, walking and balance exercises which were mainly focused on the lower limbs and improving walking. The program was applied 2 times per week for the total duration of 8 weeks.
Primary Outcome Measure Information:
Title
Timed 25-foot Walk Test
Description
The Timed 25-foot walk test (T25FW) is considered the "best characterized objective measure of walking disability and can be used across a wide range of walking disabilities". For the T25-FW, patients were instructed to walk as fast as they could in a safemanner along amarked 25-foot course. The time in seconds to complete each test was recorded, and the test was immediately repeated.
Time Frame
Change from baseline to week 8
Secondary Outcome Measure Information:
Title
Sit to Stand Test
Description
30 second Chair-Stand Test which is a reliable and valid measure used to assess lower extremity strength and endurance is used where the number of sitting and getting up within 30 seconds gives the score of the test.
Time Frame
Change from baseline to week 8
Title
Timed Up and Go test
Description
Functional mobility was evaluated using the Timed up and Go Test 'TUG' test, which is also reliable and valid test for people with Parkinson's disease. (Morris2001; Van2016). Upon issuing the command "Go," the participants stood up from a normal chair, walked 3 meters, turned, walked back to the chair, and sat. The time began with the command "Go" and ended when the participants sat back to the chair. This test was repeated three times, and the shortest performance time was recorded
Time Frame
Change from baseline to week 8
Title
Modified Ashworth Scale
Description
Modified Ashworth Scale (MAS) is one of the reliable and valid methods to measure muscle spasticity. The procedure to evaluate specific muscle groups; passively moved through the range of motion of a limb, and the resistance encountered during muscle stretch is rated on a five-point scale. Ashworth defines this rating as; 0 = no increase in tone, 1 = slight increase in tone at the end of the range of motion, 1+ = slight increase in tone throughout less than half the range of motion, 2 = increased muscle tone throughout the full range of motion, but passive movement is present. 3=tone movement that makes passive movement difficult, 4=rigidity .
Time Frame
Change from baseline to week 8
Title
Muscle Length Measurement
Description
Bilateral muscle length measurements were obtained using a standard goniometer. The patient positioned in supine position; the dorsiflexion has been tested for the gastrocnemius, the straight leg rise - hip angle has been measured to assess hamstrings, the Thomas test was used to assess iliopsoas and hip abduction angle was used for adductor group muscles.
Time Frame
Change from baseline to week 8
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of Hereditary Spastic Paraplegia at least 1 year ago
Exclusion Criteria:
Having another neurological disorder
An orthopedic deformity in the lower extremity
Having a serious cognitive impairment
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ferda Selcuk
Organizational Affiliation
European University of Lefke
Official's Role
Principal Investigator
Facility Information:
Facility Name
Dr. Burhan Nalbantoğlu State Hospital
City
Nicosia
Country
Cyprus
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Effect of Dalfampridine in Patients With Hereditary Spastic Paraplegia
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