Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders (FBIndex)

Currently, there are no standardised fall risk scores or guidelines on when to use appropriate assistive gait devices (AGDs) for people with neuromuscular disorders (NMDs). There is a clear medical unmet need to provide a battery of appropriate locomotor gait assessments to determine the risk of falling for patients with NMDs and give clear guidelines to prescribe an appropriate AGD.

The primary goal is to confirm whether the clinical battery assessments (Heel-Rise Test (HRT), Chair-Rise Test (CRT), Semi-tandem Stand (STS), Trunk-Rise Test (TRT), Foot-Tapping Test (FTT), Timed Up and Go (TUG), 10-Meter-Walk Test (10MWT) and 6-Minute-Walk Test (6MWT) can be validated and generalized to other NMD target populations that meet broader eligibility criteria based on used clinical assessments. The second objective of this project is to provide intra- and inter-observer reliability and test-retest reliability of included clinical assessments used to determine the risk of falling for patients with NMDs. Finally, all data will be compared with norm data from the healthy population (n=15) collected retrospectively.

Study Overview

• Status: Enrolling by invitation
• Conditions: Amyotrophic Lateral Sclerosis; Myasthenia Gravis; Myotonic Dystrophy; Friedreich Ataxia; Spinal Muscular Atrophy; Guillain-Barré Syndrome; Chronic Inflammatory Demyelinating Polyneuropathy; Pompe Disease; Inclusion Body Myositis; Limb-girdle and Facioscapulohumeral Muscular Dystrophies; Lambert-Eaton-Syndrome; Hereditary Motor Sensory Neuropathy
• Intervention / Treatment: Diagnostic test: FBIndex

Detailed Description

The NMD people are characterised by a complex muscle weakness caused by a combination of different factors. These include reduced endurance, lack of explosive muscle force and power, intramuscular strength coordination, and reduced balance. Those parameters need to be considered when developing an appropriate fall risk score. The combination of short muscular function and balance assessments with short clinical scores, can be a new, valid approach to evaluating the patient's risk of falling. In addition, it assists in creating a quick checkup for prescribing an appropriate assistive device.

In the first part (feasibility study) of this project, the following force plate tests were identified as suitable to assess muscle power, force and balance variables: Heel-Rise Test (HRT), Chair-Rise Test (CRT), Semi-tandem Stand (STS), Trunk-Rise Test (TRT), Foot-Tapping Test (FTT). They were used as predictors to assess patients muscle weakness and disability. In addition to muscle power and force data collected on the force plate, a second rater manually evaluated the time required to perform this test using a stopwatch. Additionally, three separate tests, Timed Up and Go (TUG), 10-Meter-Walk Test (10MWT) and 6-Minute-Walk Test (6MWT), have been evaluated separately as standard care procedures. The TUG, 10MWT and 6MWT have been used to assess remaining motor skills (endurance, walking speed and coordination) that cannot be covered by HRT, CRT, STS, TRT and FTT. As a third parameter used for correlation analysis, two risk of fall scales, Falls Efficacy Scale International (FES-I) and Morse Fall Scale (MFS), have been used. The found data from the feasibility study, which is planned to be published, suggest a moderate to moderate to strong correlation between HRT, CRT, STS, TRT, FTT, TUG, 10MWT and 6MWT and the FES-I and will be used in in the follow-up study. The MFS was excluded from the final test battery due to a lack of correlation with dependent variables. Moreover, the dependent variables assessed on the force plate, HRT, CRT, STS, TRT, and FTT (power and force), were correlated with variables collected by performing the same test using only manual time. Applying muscle strength and power data on the force plate was necessary to confirm internal consistency, content and criterion validity, as well as the degree to which the HRT, CRT, STS, TRT and FTT test can be an adequate reflection of the same test data using only manual time variables, as the final Friedrich-Baur Risk of Fall Index (FBIndex) should be conducted without the force plate using only time variables.

This study decided to include more patients and more different neuromuscular diseases to assess a wider range of muscle weaknesses that can lead to falls. Moreover, the final test battery was defined, and the standardised methodological procedure was established.

Using regression and discrimination analysis, the cross-sectional construct validity of a priori hypotheses: "Is there a multiple correlation between HRT, CRT, STS, TRT, FTT, TUG, 10MWT and 6MWT with FES-I?" will be tested.

• Study Type: Observational
• Enrollment (Estimated): 108

Contacts and Locations

Study Locations

• Germany
  • Bavaria
    • München, Bavaria, Germany, 80336
      • Friedrich-Baur-Institut, Neurologische Klinik und Poliklinik, LMU Klinikum, Ludwig-Maximilians-Universität München

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Probability Sample

Study Population

Following neuromuscular diseases confirmed by the genetic report and/or clinical final diagnosis will be included in the study: inclusion body myositis, myotonic dystrophies, limb-girdle and facioscapulohumeral muscular dystrophies, Pompe disease, myasthenia gravis, Lambert-Eaton-Syndrome, amyotrophic lateral sclerosis, spinal muscular atrophy, Guillain-Barré syndrome, chronic inflammatory demyelinating polyneuropathy, Friedreich-Ataxia, hereditary motor sensory neuropathy.

Description

Inclusion Criteria:

• Only patients with adequate cognitive and communicative function to give informed consent and to fill out the scale assessing the risk of falling will be included.

Exclusion Criteria:

• Patients who are unable to walk without AGD for at least 10 meters.
• Patients who had knee, hip or back surgery in the last three months.
• Patients who suffer from polyneuropathy or peripheral neuropathy.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Friedrich-Baur-Institute; All patients in group A will undergo single tests (cross-over design data):HRT, CRT, STS, FTT and TRT on the Leonardo Mechanograph® Ground Reaction Force Plate, assessing power and force data. Additionally, the time to perform the tests will be assessed manually. The 10MWT, 6MWT and TUG tests are performed without a ground reaction force plate and only time parameters are assessed. The group A will have an additional subgroup A1 of n=20 patients, which will be tested two times within two weeks to confirm intra-rater (n=10) and inter-rater reliability (n=10).No intervention will be carried out.	Diagnostic test: FBIndex; There will be no intervention.
Medical Park Bad Feilnbach; The group B will perform the same tests without using a force plate, assessing only the time parameters. No intervention will be carried out.	Diagnostic test: FBIndex; There will be no intervention.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Chair-Rise Test (CRT)	This is a sit-to-stand test with five repetitions. Three sets will be performed, and the fastest one will be scored. In Group A, the test uses a bench with a height of 46 cm anchored to the force plate.Only the best rise will be analyzed to collect force and power data. The main outcome parameter is the time required to perform the five repeat movements. In Group A, the examiner will assess the performance time of five sit-to-stand movements from the first to last repetition and the power data (Pmax rel. W/kg) from the force plate. In Group B, the examiner will only record the time taken to complete the test.	Immediately after the ICF is signed
Trunk Rise Test (TRT)	Participants slowly bend their knees with erect straight upper body until the fingertips touch their feet or lower leg on the inside and then cross the arms on their chest and grab their shoulders with their hands. The instructions will follow: "After the signal, rise as fast as possible, one time (movement compared to lifting a heavy object). You can use your arm to push yourself up on your thigh if you cannot get up without it". Three sets will be done, and the fastest result will be evaluated. In Groups A and B, the examiner will assess the performance time from the start position "forward bend 90°" to stand up straight and also assess whether participants use their arms or not to stand up straight. In Group A, time is assessed with power data (Pmax rel. W/kg) from the force plate. In Group B, the examiner will only record the time to perform the test on a solid concrete ground floor.	Immediately after the ICF is signed
Heel-Rise Test (HRT)	It consists of five bilateral maximal heel rises to achieve a maximal speed of upward movement. Three sets will be done, and the fastest result will be evaluated. The instructions will follow: "Rise to the tip of your toes five-times in a row as fast and high as possible. It is important that you keep your knees straight. Cross your arms on your chest and grab your shoulders with your hands. Move to your tiptoes as fast and high as possible." In Group A, the examiner will assess the performance time of five bilateral heel rises from first to last, along with the power data (P.max rel. kg- W/kg) from the force plate. In Group B, the examiner will only record the time to perform five bilateral heel rises from first to last tiptoes on a solid concrete ground floor.	Immediately after the ICF is signed
Semi-Tandem stand, eyes open (STSeo) and eyes closed (STSeo)	In this test, participants will be instructed to stand as still as possible in an upright position with both arms crossed in front of the upper body for starting, first with eyes open. Then eyes closed, in a Semi-Tandem stand as long as possible (for a maximum of 30 sec.). Three sets will be done. During the test, the Leonardo Mechanography system records the position of the center of pressure on the platform. In Group A, the time to the first drop or side-step is measured, up to a maximum of 30 seconds, together with power data (Ellip. Average cm) from the force plate. In Group B, the examiner records only the time to first drop or side-step, up to a maximum of 30 seconds. The Group B will do a semi-tandem stand on a solid concrete floor with a 1 cm wide line between the inside of the soles of the feet.	Immediately after the ICF is signed
Foot-Tapping Test (FTT) (sitting)	The FTT is designed to assess coordination and reaction time for the foot. Sitting on a 46 cm high bench, the participant's task is to tap ten times with the same foot (dorsal flexion, plantar flexion) as quickly as possible while keeping the heel on the ground. The participants were instructed to tap first with their right foot 10 times as quickly as possible. The same procedure will be repeated with the left feet, and three sets of left/right will be carried out. The participants need to sit straight, with 90° between the lower and upper leg and crossed arms over the chest. In Group A, the time for 10- foot tappings together with power data (max frequency and average contact time) from the force plate will be assessed. In Group B, the examiner records only the time. In Group B, participants are seated on a 46 cm high bench or chair with no backrest.	Immediately after the ICF is signed
10-Meter Walk Test	Walking at maximum speed will be measured in a long corridor with an even surface over 10 meters with a still-standing start and a "flying" finish to a target 2.5 meters beyond the 10 m mark. The stopwatch started with the word "Go" ("Ready-Steady-Go").	Immediately after the ICF is signed
Six-Minute Walk test	For the 6MWT, the individuals will be instructed to walk 30 m between two marks on the floor. After passing either mark, they will be told to turn and walk back. They will be instructed to walk as far as possible for six minutes and were allowed to rest (only while standing) and then to continue walking. They will be informed every minute of the remaining test time.	Immediately after the ICF is signed
Time Up and Go (TUG)	The TUG test measures the time it takes to stand up from a chair, walk three meters, turn around and sit down in the same chair. The subjects start with arm support from a seated position in a chair of normal height (44-45 cm) with arms, walk at a comfortable and safe pace to a mark on the floor 3 meters away, turn around, walk back to the chair, turn again and sit down. Time is measured from the start of the moment until participants touch the chair backrest with their back.	Immediately after the ICF is signed

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Falls Efficacy Scale-International (FES-I)	FES_I, a valid and reliable risk of fall scale used for different diagnoses, will be performed. The patient will carry out the FES-I independently. The FES-I is a scale used to measure fear of falling, self-efficacy, and balance confidence. It is suitable for use in research and clinical practice.	Immediately after the ICF is signed

Collaborators and Investigators

• Sponsor: LMU Klinikum
• Collaborators: Medical Park AG

Publications and helpful links

General Publications

• Yardley L, Beyer N, Hauer K, Kempen G, Piot-Ziegler C, Todd C. Development and initial validation of the Falls Efficacy Scale-International (FES-I). Age Ageing. 2005 Nov;34(6):614-9. doi: 10.1093/ageing/afi196.
• Podsiadlo D, Richardson S. The timed "Up & Go": a test of basic functional mobility for frail elderly persons. J Am Geriatr Soc. 1991 Feb;39(2):142-8. doi: 10.1111/j.1532-5415.1991.tb01616.x.
• Murphy SM, Herrmann DN, McDermott MP, Scherer SS, Shy ME, Reilly MM, Pareyson D. Reliability of the CMT neuropathy score (second version) in Charcot-Marie-Tooth disease. J Peripher Nerv Syst. 2011 Sep;16(3):191-8. doi: 10.1111/j.1529-8027.2011.00350.x.
• Dias N, Kempen GI, Todd CJ, Beyer N, Freiberger E, Piot-Ziegler C, Yardley L, Hauer K. [The German version of the Falls Efficacy Scale-International Version (FES-I)]. Z Gerontol Geriatr. 2006 Aug;39(4):297-300. doi: 10.1007/s00391-006-0400-8. German.
• DiPaolo G, Jimenez-Moreno C, Nikolenko N, Atalaia A, Monckton DG, Guglieri M, Lochmuller H. Functional impairment in patients with myotonic dystrophy type 1 can be assessed by an ataxia rating scale (SARA). J Neurol. 2017 Apr;264(4):701-708. doi: 10.1007/s00415-017-8399-x. Epub 2017 Feb 6.
• Runge M, Hunter G. Determinants of musculoskeletal frailty and the risk of falls in old age. J Musculoskelet Neuronal Interact. 2006 Apr-Jun;6(2):167-73.
• Dharmadasa T, Matamala JM, Huynh W, Zoing MC, Kiernan MC. Motor neurone disease. Handb Clin Neurol. 2018;159:345-357. doi: 10.1016/B978-0-444-63916-5.00022-7.

Study record dates

Study Major Dates

• Study Start (Actual): September 9, 2024
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): January 1, 2028

Study Registration Dates

• First Submitted: September 9, 2024
• First Submitted That Met QC Criteria: September 18, 2024
• First Posted (Actual): September 20, 2024

Study Record Updates

• Last Update Posted (Estimated): September 10, 2025
• Last Update Submitted That Met QC Criteria: September 3, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: Neuromuscular Disorders; Neuromuscular Diseases; Risk of falls; Risk of Falls Index; Assistive Gait Device
• Additional Relevant MeSH Terms: Post-Infectious Disorders; Musculoskeletal Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Muscular Diseases; Pathologic Processes; Neoplasms by Site; Neoplasms; Chronic Disease; Disease Attributes; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Autoimmune Diseases; Immune System Diseases; Peripheral Nervous System Diseases; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Neurodegenerative Diseases; Congenital Abnormalities; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Heredodegenerative Disorders, Nervous System; Muscular Disorders, Atrophic; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Nervous System Malformations; Motor Neuron Disease; Myositis; Polyneuropathies; Lysosomal Storage Diseases, Nervous System; Mitochondrial Diseases; Cerebellar Diseases; Polyradiculoneuropathy; Glycogen Storage Disease; Muscular Dystrophies; Myotonic Disorders; Spinocerebellar Degenerations; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathological Conditions, Signs and Symptoms; Nutritional and Metabolic Diseases; Lambert-Eaton Myasthenic Syndrome; Myotonic Dystrophy; Myasthenia Gravis; Amyotrophic Lateral Sclerosis; Muscular Atrophy, Spinal; Hereditary Sensory and Motor Neuropathy; Glycogen Storage Disease Type II; Myositis, Inclusion Body; Muscular Dystrophy, Facioscapulohumeral; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating; Neuromuscular Diseases; Friedreich Ataxia; Guillain-Barre Syndrome
• Other Study ID Numbers: 24-0636 (Other Identifier: Ludwig-Maximilians-Universität München / Ethikkommission)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED
• IPD Plan Description: Undecided: It is not yet known if there will be a plan to make IPD available.

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