Hereditary Spastic ParaplegiaNCT05613114
Effect of Dalfampridine in Patients With Hereditary Spastic Paraplegia
A study testing Dalfampridine 10 MG and Physiotherapy in people with Hereditary Spastic Paraplegia, run by European University of Lefke at 1 site in Cyprus.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hereditary Spastic Paraplegia
- What is being tested?
- Dalfampridine 10 MG and Physiotherapy
- Type of treatment
- Pill or oral medicine, Lifestyle or therapy programme
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 8 participants
- Where
- 1 siteCyprus
- Study dates
- 3 Aug 2020to 12 Mar 2021
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of Hereditary Spastic Paraplegia at least 1 year ago
Who cannot join
- Having another neurological disorder
- An orthopedic deformity in the lower extremity
- Having a serious cognitive impairment
Trial locations and contacts
1 site in 1 country.
About this study
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.
- Conditions
- Hereditary Spastic Paraplegia
- Being tested
- Dalfampridine 10 MG, Placebo, Physiotherapy
- Medical subject headings
- Spastic Paraplegia, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dalfampridine 10 MGDrug
The participants in the experimental group received dalfampridine administered as 10 mg extended-release tablets every twelve hours for 8 weeks.
PlaceboDrug
Control group received a placebo drug with the same administration method (2 times per week for the total duration of 8 weeks).
PhysiotherapyBehavioural
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.
| Study group | Role | What they receive |
|---|---|---|
| Medication | Experimental | Dalfampridine plus physiotherapy Receives: Drug: Dalfampridine 10 MG; Behavioral: Physiotherapy |
| No Medication | Placebo comparator | Placebo plus physiotherapy Receives: Drug: Placebo; Behavioral: Physiotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Change from baseline to week 8
Secondary outcomes (4)
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.
Measured: Change from baseline to week 8
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
Measured: Change from baseline to week 8
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 .
Measured: Change from baseline to week 8
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.
Measured: Change from baseline to week 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Nov 2022
- Study start3 Aug 2020
- Primary completion12 Mar 2021
- Study completion12 Mar 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hereditary Spastic ParaplegiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Outcomes assessor
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- European University of Lefke
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ferda Selcuk, European University of Lefke
- Investigators
- Ferda Selcuk, Principal investigator, European University of Lefke
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05613114
- Sponsor's protocol ID
- 45/20
- Status last verified
- Nov 2022
- First submitted
- 4 Nov 2022
- Last updated
- 22 Nov 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Selcuk Muhtaroglu F, Belgen Kaygisiz B, Usar Incirli S, Kahraman T. Dalfampridine as a promising agent in the management of hereditary spastic paraplegia: A triple-blinded, randomized, placebo-controlled pilot trial. J Clin Neurosci. 2023 Nov;117:136-142. doi: 10.1016/j.jocn.2023.09.026. Epub 2023 Oct 5.
PubMed 37804674
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Dalfampridine in Patients With Hereditary Spastic Paraplegia
- ClinicalTrials.gov ID
- NCT05613114
- Acronym / sponsor ID
- 45/20
- Conditions
- Hereditary Spastic Paraplegia
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- European University of LefkeUniversity, hospital or other
- Study start
- 3 Aug 2020 (actual)
- Primary completion
- 12 Mar 2021 (actual)
- Study completion
- 12 Mar 2021 (actual)
- First posted
- 14 Nov 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Medication | Experimental | Drug: Dalfampridine 10 MG, Behavioral: PhysiotherapyDalfampridine plus physiotherapy |
| No Medication | Placebo comparator | Drug: Placebo, Behavioral: PhysiotherapyPlacebo plus physiotherapy |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Diagnosis of Hereditary Spastic Paraplegia at least 1 year ago
Key exclusion criteria (3 listed)
- Having another neurological disorder
- An orthopedic deformity in the lower extremity
- Having a serious cognitive impairment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Dalfampridine 10 MGThe participants in the experimental group received dalfampridine administered as 10 mg extended-release tablets every twelve hours for 8 weeks. | Drug | Medication |
| PlaceboControl group received a placebo drug with the same administration method (2 times per week for the total duration of 8 weeks). | Drug | No Medication |
| PhysiotherapyConventional 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. | Behavioural | Medication, No Medication |
Primary outcome
Timed 25-foot Walk Test
Time frame: Change from baseline to week 8
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.
4 secondary outcomes
Sit to Stand Test
Time frame: Change from baseline to week 8
Timed Up and Go test
Time frame: Change from baseline to week 8
Modified Ashworth Scale
Time frame: Change from baseline to week 8
Muscle Length Measurement
Time frame: Change from baseline to week 8
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Nov 2022.
- Derived from this study: PubMed 37804674Selcuk Muhtaroglu F, Belgen Kaygisiz B, Usar Incirli S, Kahraman T. Dalfampridine as a promising agent in the management of hereditary spastic paraplegia: A triple-blinded, randomized, placebo-controlled pilot trial. J Clin Neurosci. 2023 Nov;117:136-142. doi: 10.1016/j.jocn.2023.09.026. Epub 2023 Oct 5.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ferda Selcuk principal investigator
European University of Lefke
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Dalfampridine 10 MG and Physiotherapy in people with Hereditary Spastic Paraplegia, run by European University of Lefke at 1 site in Cyprus.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Cyprus. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Timed 25-foot Walk Test (time frame: Change from baseline to week 8). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05613114, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Nov 2022.