Physiotherapy in Hereditary Spastic Paraplegia

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

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.

Study Overview

• Status: Completed
• Conditions: Hereditary Spastic Paraplegia
• Intervention / Treatment: Behavioral: physiotherapy

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 53
• Phase: Phase 2

Contacts and Locations

Study Locations

• Germany
  • Tübingen, Germany, 72076
    • University Hospital Tübingen, Center for Neurology

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Tübingen physiotherapy concept; Will receive our therapy concept and perform self-trainings on a regular basis.	Behavioral: physiotherapy; 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.
No Intervention: controls; 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?

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

Collaborators and Investigators

• Sponsor: University Hospital Tuebingen

Publications and helpful links

General Publications

• Schule R, Holland-Letz T, Klimpe S, Kassubek J, Klopstock T, Mall V, Otto S, Winner B, Schols L. The Spastic Paraplegia Rating Scale (SPRS): a reliable and valid measure of disease severity. Neurology. 2006 Aug 8;67(3):430-4. doi: 10.1212/01.wnl.0000228242.53336.90.

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2015
• Primary Completion (Actual): March 30, 2017
• Study Completion (Actual): March 30, 2017

Study Registration Dates

• First Submitted: May 20, 2019
• First Submitted That Met QC Criteria: May 22, 2019
• First Posted (Actual): May 23, 2019

Study Record Updates

• Last Update Posted (Actual): May 23, 2019
• Last Update Submitted That Met QC Criteria: May 22, 2019
• Last Verified: May 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neurologic Manifestations; Congenital Abnormalities; Genetic Diseases, Inborn; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Manifestations; Heredodegenerative Disorders, Nervous System; Nervous System Malformations; Paralysis; Muscle Hypertonia; Polyneuropathies; Hereditary Sensory and Motor Neuropathy; Muscle Spasticity; Paraplegia; Spastic Paraplegia, Hereditary
• Other Study ID Numbers: Physiotherapy in HSP

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No
• IPD Plan Description: Available upon request

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No