A Pilot Study of Shockwave Therapy in HSP

A Pilot Study of Shockwave Therapy for Lower Extremity Spasticity in Persons With Genetically-Confirmed HSP

This is a pilot study of the efficacy, safety, and tolerability of shockwave therapy for the treatment of spasticity in Hereditary Spastic Paraplegia.

Study Overview

• Status: Withdrawn
• Conditions: Hereditary Spastic Paraplegia
• Intervention / Treatment: Device: Shockwave Therapy

Detailed Description

This is a pilot study investigating the efficacy, safety, and tolerability of Extracorporeal Shockwave Therapy (ESWT) for the treatment of lower-extremity spasticity in the setting of Hereditary Spastic Paraplegia (HSP). 8 participants will be treated with shockwave therapy targeting sites of maximal spasticity in the lower extremities for 3 sessions at a rate of once per week. Participants will fill out baseline questionnaires assessing their overall wellbeing and function prior to treatment session 1, and will complete follow-up questionnaires at visit 2, visit 3, a phone call within 7 days of visit 3, and 8 weeks after initial visit. Total time enrolled in the study will be 8 weeks

• Study Type: Interventional
• Phase: Not Applicable

Contacts and Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

1. Over the age of 18
2. Diagnosis of genetically-confirmed HSP
3. Score of 2, 3 or 4 on the PLSFRS walking question
4. 30 days stable dosages of oral spasticity medications prior to screening and throughout study participation
5. Able to come to site for treatment sessions
6. Able to understand all study procedures

Exclusion Criteria:

1. Diagnosis of any other neurological disorder that may impact gait
2. Lower motor neuron (LMN) disease or combined UMN and LMN
3. Non-ambulatory or requiring two-person assistance for any functional ambulation on most days
4. Less than 3 months of symptoms
5. Have received SWT within the past 3 months
6. Prior botulinum toxin injection within 3 months or planning to receive botulinum toxin injections during the study participation for spasticity management
7. Presence of an intra-thecal baclofen pump
8. Women who are pregnant. This is due to the fact that pregnancy will reduce participants activity after the treatment and the safety concerns related to shockwave treatment.
9. Patients with neuropathy affecting sensation to pain
10. Patients with a known underlying cardiac disease that could be affected by shockwave therapy
11. Concomitant intake of another clinical trial or other off-label experimental treatments that might influence study outcomes
12. Presence of unstable psychiatric disease, cognitive impairments, or dementia impairing study participation
13. Presence of other chronic neurological diseases such as Alzheimer's Parkinson's, stroke, or multiple sclerosis
14. Personal history of narcotic overuse for chronic pain or substance abuse or dependence
15. Contraindications for ESWT, including local skin breakdown, infection, malignancy or other soft tissue pathology at sites of planned treatment.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment Group; Pilot group of all 8 anticipated participants	Device: Shockwave Therapy; Participants will receive shockwave therapy once per week for three weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Numerical Rating Scale for Pain	A rating of pain from 0 (none) to 10 (worst)	0-8 weeks; assessed at baseline visit, 1, 2, 3, and 8 weeks
Primary Lateral Sclerosis Functional Rating Scale (PLSFRS)	The score of the rating scale ranges from 0-68, where 68 is a person who is completely asymptomatic.	0-8 weeks; assessed at baseline visit, 1, 3, and 8 weeks
Neurological Quality of Life (Neuro-QoL) Questionnaire: Fatigue Short Form	The score of this questionnaire ranges from 8 (least fatigue) to 40 (most fatigue)	0-8 weeks; assessed at baseline visit, 1, 3, and 8 weeks
Foot and Ankle Ability Measure (FAAM) Questionnaire: Activities of Daily Living Subscale	The score of this questionnaire ranges from 0 (maximally symptomatic in activities of daily living) to 84 (asymptomatic in activities of daily living).	0-8 weeks; assessed at baseline visit, 1, 3, and 8 weeks
Global Rate of Change (GROC) Questionnaire	The score ranges from -7 (maximally negative change) to +7 (maximally positive change)	0-8 weeks; assessed at baseline visit, 1, 3, and 8 weeks
Neurological Quality of Life (Neuro-QoL) Questionnaire: Lower Extremity Function - Mobility Short Form	The score of this questionnaire ranges from 8 (minimal lower extremity function) to 40 (maximal lower extremity function)	0-8 weeks; assessed at baseline visit, 1, 3, and 8 weeks
Neurological Quality of Life (Neuro-QoL) Questionnaire: Positive Affect and Wellbeing Short Form	The score of this questionnaire ranges from 9 (minimal wellbeing) to 45 (maximal wellbeing)	0-8 weeks; assessed at baseline visit, 1, 3, and 8 weeks
Neurological Quality of Life (Neuro-QoL) Questionnaire: Satisfaction with Social Roles and Activities Short Form	The score of this questionnaire ranges from 8 (minimal satisfaction) to 40 (maximal satisfaction)	0-8 weeks; assessed at baseline visit, 1, 3, and 8 weeks
Foot and Ankle Ability Measure (FAAM) Questionnaire: Sports Subscale	The score of this questionnaire ranges from 0 (maximally symptomatic in sports activities) to 32 (asymptomatic in sports activities)	0-8 weeks; assessed at baseline visit, 1, 3, and 8 weeks
Ten-Meter Walk Test	Participants will be asked to walk ten meters 2x at their self-selected walking speed and 2x at their fast walking speed. Time taken to complete walks will be measured.	0-3 weeks; assessed at baseline visit, 1, 2, and 3 weeks

Collaborators and Investigators

• Sponsor: Spaulding Rehabilitation Hospital
• Collaborators: Massachusetts General Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): January 1, 2023
• Primary Completion (Estimated): October 15, 2023
• Study Completion (Estimated): December 15, 2023

Study Registration Dates

• First Submitted: May 27, 2022
• First Submitted That Met QC Criteria: June 6, 2022
• First Posted (Actual): June 9, 2022

Study Record Updates

• Last Update Posted (Actual): February 8, 2024
• Last Update Submitted That Met QC Criteria: February 6, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neurologic Manifestations; Congenital Abnormalities; Genetic Diseases, Inborn; Neuromuscular Diseases; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Nervous System Malformations; Paralysis; Polyneuropathies; Hereditary Sensory and Motor Neuropathy; Paraplegia; Spastic Paraplegia, Hereditary
• Other Study ID Numbers: 2022P000682

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: Yes
• product manufactured in and exported from the U.S.: Yes