The Risk of Falls Index for Patients With Neuromuscular Disorders

The FBIndex to Determine the Risk of Falls and Its Translation to Assistive Gait Device Care for Patients With Neuromuscular Disorders

The combination of short quantitatively assessing muscular function and balance in combination with short clinical scores, can be a new valid approach to evaluate the patient risk of fall and help to create a quick checkup test to prescribe an appropriate assistive device.

The primary goal of this project is to provide a short battery of clinical assessments used to determine risk of falling for patients with neuromuscular diseases (NMD) based on correlation between clinical assessments between two groups of NMD patients and scales used to assess risk of falling for patients.

Study Overview

• Status: Recruiting
• Conditions: Myasthenia Gravis; Myotonic Dystrophy; Inclusion Body Myositis; Facioscapulohumeral Muscular Dystrophies; The Falls Efficacy Scale International; The Morse Fall Scale
• Intervention / Treatment: Diagnostic test: The risk of falls

Detailed Description

1. Introduction Currently, we could not find standardized risk of fall score or guidelines as when to use appropriative assistive gait devices (AGD) for patients with neuromuscular disorder/diseases (NMDs). There is a clear medical unmet need to provide a battery of appropriate locomotor gait assessments used to determine the risk of falling for patients with NMDs and give clear guidelines requisite to prescribe an appropriate AGD. Furthermore, patients and medical workers are in a constant struggle with health agencies in order to timely receive appropriate AGD. The AGD prescription that obtain a standardized and evidence-based patient status score can support and simplify this process.
2. Methods A feasibility pilot study protocol with one cohort group was constructed. The study protocol follow the PICO Consort criteria and will be registered at ClinicalTrials.gov. All participants will sign a written consent. The study will be approved by ethic committee of the Ludwig-Maximilians-Universität München. The pilot group (n=30) will be examinant as part of annual routine examination in the Friedrich-Baur-Institute at the Department of Neurology, Munich, Germany. Following neuromuscular diseases are included in the study: inclusion body myositis, myotonic dystrophies, limb girdle and facioscapulohumeral muscular dystrophies, and myasthenia gravis. The patient data will be collected for one year. After the participants has been collected, the statistical data analysis will be carried out to provide power analysis and to adjust the inclusion criteria if needed. If the found data are insufficient, an additional 10 subjects per group will be added.

3. Outcomes The descriptive data about age, age of onset, age of first symptoms and time of symptoms will be assessed. The pilot group will be preforming two test batteries, plus two scores.

   Test battery one (TB1) includes : Heel-Rise-Test (HRT), Chair-Rise Test (CRT), Semi-tandem stand (STS), Trunk Rise Test (TRT), and Foot-tapping speed (FTS). Test battery two (TB2) includes Timed Up and Go test, 10 Meter Walk Test and Six Minute Walk test.

4. Inclusion and Exclusion Criteria The patients between 18- and 65-year-old will be included in the study. Only patients with adequate cognitive and communication function to give informed consent and to fill out the scales assessing risk of falling will be included. Excluded are patients which are not able to perform motoric functional assessments without assistive gait device. The NMD needs to be confirmed by genetic report and/or disease deepening neurophysiological examination.
5. Study purpose and the project perspective The study is part of a larger project made by Friedrich-Baur-Institute to clarify the patient and therapist possibilities by selecting AGD, and to help the physician to prescribe the right AGD. Nevertheless, this study builds on an ongoing systematic review; "The assistive gait technology devices and their implementation in activities of daily living for patients with neuromuscular disease". The purpose of review is to evaluate appropriative use of AD as evaluated benefit and tolerability of current AGD supporting lower limbs functions during activities of daily living for individuals with neuromuscular disorders.

Due to the fact that currently in Germany, no general standardized risk of fall index for patient with NMDs was found, there is a clear unmet medical to provide a battery of appropriate locomotor gait tests used to determine risk of falling and generally to explore this topic in more detail. The final goal is to test index battery on its reliability in clinical setting without ground reaction force plate. The index should be ability to quickly assess the clinical status of the patient without use of expensive and time consuming tools to assess risk of fall.

• Study Type: Observational
• Enrollment (Estimated): 30

Contacts and Locations

• Study Contact: Name: Corinna Wirner-Piotrowski; Email: Corinna.Wirner@med.uni-muenchen.de
• Study Contact Backup: Name: Marko Mijic; Phone Number: +4989440057078; Email: marko.mijic@med.uni-muenchen.de

Study Locations

• Germany
  • Bavaria
    • München, Bavaria, Germany, 80336
      • Recruiting
      • Marko Mijic
      • Contact: Corinna Wirner-Piotrowski; Email: Corinna.Wirner@med.uni-muenchen.de
      • Contact: Marko Mijic; Email: marko.mijic@med.uni-muenchen.de

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Non-Probability Sample

Study Population

Following neuromuscular diseases are included in the study: inclusion body myositis, myotonic dystrophies, limb girdle and facioscapulohumeral muscular dystrophies, and myasthenia gravis.

Description

Inclusion Criteria:

• Patients between 18- and 65-year-old
• Patients with adequate cognitive and communication function to fill out the scales assessing risk of falling
• The neuromuscular disease needs to be confirmed by genetic report and/or disease deepening neurophysiological examination.

Exclusion Criteria:

- Patients which are not able to perform motoric functional assessments without assistive gait device.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Neuromuscular disorders	Diagnostic test: The risk of falls; Heel-Rise-Test, Chair-Rise Test, Semi-tandem stand, Trunk Rise Test, and Foot-tapping speed. Test battery two includes Timed Up and Go test, 10 Meter Walk Test, Six Minute Walk test, The Falls Efficacy Scale International FES-I, The Morse Fall Scale.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
FBIndex	The primary goal of this project is to provide a short battery of clinical assessments used to determine risk of falling for patients with NMDs based on correlation between clinical assessments: Heel-Rise-Test, Chair-Rise Test, Semi-tandem stand, Trunk Rise Test, and Foot-tapping speed, Timed Up and Go test, 10 Meter Walk Test and Six Minute Walk test, between two groups of NMD patients and scales: "The Falls Efficacy Scale International FES-I" and "The Morse Fall Scale" used to assess risk of falling for patients.	2 years

Collaborators and Investigators

• Sponsor: LMU Klinikum

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2023
• Primary Completion (Estimated): September 1, 2025
• Study Completion (Estimated): September 1, 2025

Study Registration Dates

• First Submitted: May 26, 2023
• First Submitted That Met QC Criteria: May 26, 2023
• First Posted (Actual): June 6, 2023

Study Record Updates

• Last Update Posted (Actual): October 11, 2023
• Last Update Submitted That Met QC Criteria: October 9, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Immune System Diseases; Neoplasms; Autoimmune Diseases of the Nervous System; Autoimmune Diseases; Neoplasms by Site; Genetic Diseases, Inborn; Musculoskeletal Diseases; Muscular Diseases; Neurodegenerative Diseases; Nervous System Neoplasms; Muscular Disorders, Atrophic; Heredodegenerative Disorders, Nervous System; Paraneoplastic Syndromes, Nervous System; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Myotonic Disorders; Muscular Dystrophies; Myasthenia Gravis; Myositis; Myotonic Dystrophy; Muscular Dystrophy, Facioscapulohumeral; Neuromuscular Diseases; Myositis, Inclusion Body
• Other Study ID Numbers: FBIndex_80336

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