Development and Validation of the FBIndex to Determine the Risk of Falls for Patients with Neuromuscular Disorders

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Observational
Eligible Ages 18 Years - 65 Years
Gender All
More Inclusion & Exclusion Criteria

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.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT06605612
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

LMU Klinikum
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Other, Industry
Overall Status Enrolling by invitation
Countries Germany
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Inclusion Body Myositis, Myotonic Dystrophy, 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
Additional Details

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.

Arms & Interventions

Arms

: 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.

: 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.

Interventions

Diagnostic Test: - FBIndex

There will be no intervention.

Contact a Trial Team

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International Sites

München, Bavaria, Germany

Status

Address

Friedrich-Baur-Institut, Neurologische Klinik und Poliklinik, LMU Klinikum, Ludwig-Maximilians-Universität München

München, Bavaria, 80336

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