A Pilot Study of Shockwave Therapy in HSP

Study Overview
=================
Brief Summary
-----------------
This is a pilot study of the efficacy, safety, and tolerability of shockwave therapy for the treatment of spasticity in Hereditary Spastic Paraplegia.

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

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

Conditions
-----------------
Hereditary Spastic Paraplegia

Intervention / Treatment
-----------------
* Device: Shockwave Therapy


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Over the age of 18 Diagnosis of genetically-confirmed HSP Score of 2, 3 or 4 on the PLSFRS walking question 30 days stable dosages of oral spasticity medications prior to screening and throughout study participation Able to come to site for treatment sessions Able to understand all study procedures Exclusion Criteria: Diagnosis of any other neurological disorder that may impact gait Lower motor neuron (LMN) disease or combined UMN and LMN Non-ambulatory or requiring two-person assistance for any functional ambulation on most days Less than 3 months of symptoms Have received SWT within the past 3 months Prior botulinum toxin injection within 3 months or planning to receive botulinum toxin injections during the study participation for spasticity management Presence of an intra-thecal baclofen pump 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. Patients with neuropathy affecting sensation to pain Patients with a known underlying cardiac disease that could be affected by shockwave therapy Concomitant intake of another clinical trial or other off-label experimental treatments that might influence study outcomes Presence of unstable psychiatric disease, cognitive impairments, or dementia impairing study participation Presence of other chronic neurological diseases such as Alzheimer's Parkinson's, stroke, or multiple sclerosis Personal history of narcotic overuse for chronic pain or substance abuse or dependence Contraindications for ESWT, including local skin breakdown, infection, malignancy or other soft tissue pathology at sites of planned treatment.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
Accepts Healthy Volunteers

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

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

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 |