Physiotherapy in Hereditary Spastic Paraplegia

Study Overview
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Brief Summary
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Goal of this study is to develop and evaluate a physiotherapy concept that is focused on bilateral leg spasticity and aims to reduce spastic gait disturbance and to improve mobility in patients suffering from HSP.

Detailed Description
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This is a randomized single-center parallel study with a control group design. A blinded block randomization in groups of four was performed in equal parts toward the treatment (TPC) or the control (regular standard-of-care) group using randomization.com. Group assignment was kept in a closed envelope which was opened by the participant at the end of the baseline visit. Therefore both, the movement disorder specialist as well as the physiotherapist, were blinded to the randomization result for the baseline assessment up to the beginning of the first physiotherapy training session. Therapeutic effects were evaluated at follow-up visits after 12 weeks (first visit, short-term effect) and after 26 weeks (second visit, long-term effect) comparing standard-of care with the TPC. Clinical evaluation is stated in detail below. Primary outcome assessment of this study was the change between baseline and follow-up assessment in treatment to control group in the spastic paraplegia rating scale (SPRS) score as a validated measure of disease severity (see Reference). Secondary outcome assessment was the change in walking distance in the Three-Minute-Walking test (3MW). Further exploratory outcome assessments were evaluated as effect sizes as specified below.

Official Title
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Design and Validation of a Modular Physiotherapy Concept for the Treatment of Hereditary Spastic Spinal Paralysis (HSP) - a Randomized Study

Conditions
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Hereditary Spastic Paraplegia

Intervention / Treatment
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* Behavioral: physiotherapy


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: clinical diagnosis of hereditary spastic paraplegia manifest spastic gait disturbance remaining walking ability of at least 100m in three minutes, no botulinum toxin treatment during the entire study and three months prior to study inclusion no functional electronic stimulation during the study period. Exclusion Criteria: see above

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Tübingen physiotherapy concept<br>Will receive our therapy concept and perform self-trainings on a regular basis. | Behavioral: physiotherapy<br>* Will receive two trainings. First training in week one after baseline assessment for three consecutive days 60 min twice daily by trained physiotherapists and in week three further two days with 60min once daily.<br>|
| No Intervention: controls<br>Will receive standard-care which includes their regular physiotherapy as provided by the local therapist and can include self-trainings as well. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in total Spastic paraplegia rating scale (SPRS) score | Change in the total SPRS score (see references for publication) (range 0-52 points, higher points indicated stronger disease severity) will be evaluated at two timepoints compared to baseline (12 weeks as short-term measure and 26 weeks as long-term measure). | 12 weeks and 26 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Three Minute Walk | Change in walking distance within three minutes will be assessed two timepoints compared to baseline (12 weeks as short-term measure and 26 weeks as long-term measure). | 12 weeks and 26 weeks |