Evaluation of an Intensive Training Program for Patients with Hereditary Spastic Paraparesis SPG4/Spast (WALK-up)

Hereditary spastic paraparesis is a group of inherited neurological diseases. Only symptomatic treatments exist for the moment. The Modifspa study (cf citation) carried out by the team showed that patients perceived a feeling of effectiveness of physiotherapy on lower limb spasticity. The aim of the Walk-up study is to objectivize this feeling of efficacy on gait disorders in these patients.

This is an interventional study using physical training. The study is prospective, open, randomized in 2 parallel groups, one of which is a control group. Analyses will be comparative between the 2 groups during the course of the study.

Study Overview

• Status: Recruiting
• Conditions: Hereditary Spastic Paraparesis
• Intervention / Treatment: Other: intensive reeducation

Detailed Description

Following an initial study carried out by the team (cf citation), physiotherapy appeared to be the most useful therapy for coping with spasticity, particularly when practised at least 3 times a week. The hypothesis is that this feeling experienced by patients is accurate, and that more frequent physiotherapy (3 additional sessions/week) significantly improves patients' walking speed, with a functional objective.

The main aim of the WALK-UP study is to evaluate the efficacy of a 6-week intensive physical rehabilitation program on walking speed in patients with SPG4 / SPAST-HSP. This is the most frequent genotype in Hereditary Spastic Paraparesis. All patients included in the study will receive at least one physiotherapy session per week in a liberal practice.

• Study Type: Interventional
• Enrollment (Estimated): 50
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Pauline Lallemant, MD; Phone Number: +33 1. 57.27.46.69; Email: pauline.lallemant@icm-institute.org
• Study Contact Backup: Name: Rania Hilab, CRA; Phone Number: +33 1. 57.27.46.91; Email: rania.hilab@icm-institute.org

Study Locations

• France
  • Paris, France, 75013
    • Recruiting
    • ICM, Hôpital Pitié-Salpêtrière
    • Contact: Pauline Lallemant, MD; Phone Number: +33 1. 57.27.46.69; Email: pauline.lallemant@icm-institute.org
    • Contact: Rania Hilab, CRA; Phone Number: +33 1. 57.27.46.91; Email: rania.hilab@icm-institute.org
    • Contact: Pauline Lallemant, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Patient with molecular diagnosis of hereditary spastic paraparesis based on pathogenic variant of SPAST gene,
• Walking possible for 6 minutes without human assistance (one or more technical aids are authorized: e.g. cane, walker, orthoses),
• At least 1 physiotherapy session per week already in place.
• Understanding of the protocol
• Possibility of connecting to the Internet from home to access video material provided as part of the protocol.

Exclusion Criteria:

• Botulinum toxin injection within 2 months of protocol inclusion
• Discontinuation of private physiotherapy,
• Refusal to participate in the protocol,
• Participation in another interventional clinical trial evaluating a health product or in a randomized clinical trial
• Pregnant women
• Not affiliated to a social security scheme or beneficiary of such a scheme
• Patient under guardianship or trusteeship

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: the reeducation group; The population included in the reeducation group will benefit, in addition to their usual physiotherapy care (at least 1 session per week), from : 1 group physiotherapy session per week at the ICM or by videoconference from home for 6 weeks; 2 sessions per week at home with video support sent by e-mail for 6 weeks These 3 additional video or group sessions will last 30 min. These sessions will be created and adjusted by a physiotherapist and a Physical and Rehabilitation Medicine doctor. They will include exercises such as : Stretching; posture; Muscle strengthening; Proprioception And will be performed in standing, sitting and lying positions.	Other: intensive reeducation; 1 group physiotherapy session per week at the ICM or by videoconference from home for 6 weeks; 2 sessions per week at home with video support sent by e-mail for 6 weeks
No Intervention: The control group; usual physiotherapy care (at least 1 session per week)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
to compare the before-and-after variation in distance covered in 6 minutes between the intensive physical rehabilitation group and the control group.	6-minute walk test carried out in the presence of a clinician trained in this test. The patient walks for 6 minutes on a circuit of known distance, identical for each patient. The clinician times the walking time and measures the distance covered during 6 minutes. This test is performed at inclusion and again at 6 weeks	6 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
compare the before-after variation in distance covered in 6 minutes	6-minute walk test carried out in the presence of a clinician trained in this test. This test is performed at inclusion and again at 18 weeks	18 weeks
compare the before-after variation in walking speed over a 10-meters test	10-meters test carried out in the presence of a clinician trained in this test. This test is performed at inclusion and again at 18 weeks	18 weeks
compare the clinical evolution of spasticity	clinical evolution of spasticity using Spastic Paraplegia Rating Scale (SPRS)	18 weeks
compare the lower-limb joint amplitudes	clinical evolution of lower-limb joint amplitudes using the modified Aschworth scale	18 weeks
compare the patients' mood evolution	patients' mood evolution using the Hospital Anxiety and Depression (HAD) scale	18 weeks
compare the evolution of cognitive disorders	evolution of cognitive disorders using the Cerebellar Cognitive Affective/Schmahmann Syndrome Scale (CCAS)	18 weeks
compare the before-after variation in the daily number of steps	before-after variation in the number of steps taken daily under ecological conditions, reported by pedometer	18 weeks
compare the before-after variation in heart rate	before-after variation in heart rate during walking assessments (6-minute and 10-meter tests)	18 weeks
compare the frequency and intensity of vesico-sphincter disorders	compare the frequency of vesico-sphincter disorders using Neurogenic Bowel Dysfunction sore	18 weeks
compare the anorectal disorders	compare the intensity of vesico-sphincter disorders using Urinary Symptom Profile	18 weeks
compare the quality of life	compare the quality of life using the EQ5D scale. The EQ-5D is a self-administered questionnaire that measures five dimensions of health: mobility, self-care, usual activities, pain/discomfort and anxiety/depression.	18 weeks
compare the fatigue	compare the fatigue using the Modified Fatigue Impact Scale (MFIS)	18 weeks

Collaborators and Investigators

• Sponsor: Assistance Publique - Hôpitaux de Paris

Study record dates

Study Major Dates

• Study Start (Actual): April 4, 2024
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): October 1, 2026

Study Registration Dates

• First Submitted: December 13, 2023
• First Submitted That Met QC Criteria: January 19, 2024
• First Posted (Actual): January 29, 2024

Study Record Updates

• Last Update Posted (Estimated): November 25, 2024
• Last Update Submitted That Met QC Criteria: November 21, 2024
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Keywords: physical rehabilitation program; hereditary spastic paraparesis
• Additional Relevant MeSH Terms: Neurologic Manifestations; Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Muscle Hypertonia; Neuromuscular Manifestations; Paresis; Muscle Spasticity; Paraparesis; Paraparesis, Spastic
• Other Study ID Numbers: APHP230987

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No