Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders

NCT ID: NCT04907162

Last Updated: 2022-08-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

82 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-04-15

Study Completion Date

2022-08-15

Brief Summary

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The primary aim is to characterize the prevalence, severity and quality of musculoskeletal nociceptive pain in adult patients with neuromuscular disorders (NMD). The secondary objectives are to evaluate whether severity and distribution of muscle pain is associated with muscle function, and to assess whether muscle pain is associated with alterations of muscle elasticity and muscle stiffness. Results of patients with neuromuscular disorders will be compared to age- and gender-matched healthy volunteers. Approx. 70 patients with neuromuscular disorders and 20 healthy volunteers will be enrolled, including patients with the following neuromuscular disorders: histologically confirmed inclusion body myositis (IBM), genetically confirmed late-onset Pompe disease (LOPD), genetically confirmed spinal muscular atrophy type 3 (SMA3), genetically confirmed facio-scapulo-humeral muscle dystrophy (FSHD), genetically confirmed myotonic dystrophy type 1 or type 2 (DM1, DM2). The duration of patient recruitment will be around 12 months.

Detailed Description

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The explorative, cross-sectional low-interventional pilot study evaluates the prevalence, severity and quality of musculoskeletal nociceptive pain in participants with defined neuromuscular disorders (NMD). Adult participants with the following neuromuscular disorders will be included: histologically confirmed inclusion body myositis (IBM), genetically confirmed late-onset Pompe disease (LOPD), genetically confirmed spinal muscular atrophy type 3 (SMA3), genetically confirmed facio-scapulo-humeral muscle dystrophy (FSHD) and genetically confirmed myotonic dystrophy type 1 or type 2 (DM1, DM2). 20 healthy participants will be enrolled as a control group. The Beck depression inventory fast screen (BDI-FS) will be used as a screening. If there is a possibility of major depression (with a BDI ≥4), patients will be excluded from the study. So only patients with BDI-FS score ≤3 at screening will be enrolled. Patients will be asked to complete the following validated disease-related and quality-of-life questionnaires: German Pain Inventory (module A and abbreviated module S and L and V), Brief Pain Inventory (BPI) and Fatigue Severity and Disability Scale (FSS). Demographic and disease related data will be obtained. A neuromuscular examination will be conducted. A clinical evaluation of muscle strength using the MRC-Scale (Medical Research Council-Scale) will be performed on both sides deltoid muscles, biceps brachii muscles, triceps brachii muscles, hip flexors, hip extensors, quadriceps femoris muscles, foot extensor and foot flexor muscles as well as axial muscles and neck flexors and extensors. The Quick Motor Function Test (QMFT) with 16 items will be performed to assess muscle and movement functions of the participants. To ensure a high level of objective measurement, muscle strength will also be assessed by using handheld dynamometry. The following muscle groups will be tested: arm abduction, elbow flexion, elbow extension, hip flexion, hip extension, knee extension, knee flexion, foot extension, foot flexion. A six-minute-walk test (6MWT) will be performed once. Additionally, first signs of muscle pain or muscle cramps will be recorded (including the quality and intensity of pain). The Borg scale to rate dyspnea will be administered before starting the 6MWT and after completing the 6MWT. For diagnosis of myofascial pain, a Pressure Pain Threshold test by using a pressure algometer is included for the trapezius, deltoid and supraspinatus muscles, the rectus femoris muscles, and the tibialis anterior muscles until the patient feels any sensation of pain. Measurement of muscle stiffness, muscle tone, relaxation periods and viscoelasticity of selected muscles will be assessed by a myotonometer. Data collected in this study will be reported using summary tables, figures, and patient data listings. Differences between the patients and the healthy volunteers will be analyzed.

Conditions

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Pompe Disease (Late-onset) Myotonic Dystrophy Type 1 (DM1) Myotonic Dystrophy Type 2 Spinal Muscular Atrophy Type 3 Inclusion Body Myositis, Sporadic Facioscapulohumeral Muscular Dystrophy 1 Healthy

Study Design

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Observational Model Type

CASE_CONTROL

Study Time Perspective

PROSPECTIVE

Study Groups

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Neuromuscular disease (NMD) Patients

The patient has one of the following neuromuscular diagnoses:

1. histologically (muscle biopsy) confirmed inclusion body myositis (IBM), or
2. genetically confirmed late-onset Pompe disease (LOPD), or
3. genetically confirmed spinal muscular atrophy type 3 (SMA3), or
4. genetically confirmed myotonic dystrophy type 1, or
5. genetically confirmed myotonic dystrophy type 2, or
6. genetically confirmed facio-scapulo-humeral muscle dystrophy (FSHD).

Beck depression inventory fast screen

Intervention Type DIAGNOSTIC_TEST

Beck depression inventory fast screen questionnaire to detect severe depression for eligibility.

German Pain Inventory

Intervention Type DIAGNOSTIC_TEST

German Pain Inventory questionnaire for evaluation of pain. Module A, abbreviated questions of module S (sociodemographic questions S1, S2, S3, S4, S5 and S8) and module L (quality of life) and V (therapies) will be used.

Brief Pain Inventory

Intervention Type DIAGNOSTIC_TEST

Validated questionnaire for pain.

Fatigue Severity and Disability Scale (FSS)

Intervention Type DIAGNOSTIC_TEST

Validated questionnaire for perceived fatigue

Quick Motor Function Test

Intervention Type DIAGNOSTIC_TEST

An evaluator observes the performance of a patient and scores the items separately on a 5-point ordinal scale (ranging from 0 to 4). A total score is obtained by adding the scores of all items and ranges between 0 and 64 points.

Handheld Dynamometry (HHD)

Intervention Type DIAGNOSTIC_TEST

To ensure a high level of objective measurement, muscle strength will also be assessed by handheld dynamometry. The following muscle groups will be tested: Arm abduction, elbow flexion, elbow extension, hip flexion, hip extension, knee extension, knee flexion, foot extension, foot flexion.

Six-minute walk test (6MWT)

Intervention Type DIAGNOSTIC_TEST

It is a sub-maximal exercise test used to assess aerobic capacity and endurance. The distance covered over a time of 6 minutes is used as the outcome by which to compare changes in performance capacity.

Pressure pain threshold

Intervention Type DIAGNOSTIC_TEST

For diagnosis of myofascial pain, pressure algometers are designed and conventionally used to measure deep pressure pain thresholds or tenderness resistance (Park, Kim et al. 2011), and the reliability of pressure pain thresholds according to raters or measurement frequencies has been proven to be relatively high (Chung, Um et al. 1992). The threshold is then determined as the arithmetic mean of the 3 series (in kPa). The measurement will be stopped immediately as the patient feels sensations of "burning", "stinging", "drilling" or "aching. Pressure algometry measurements will be performed on the trapezius, deltoid and supraspinatus muscles, the rectus femoris muscles, and the tibialis anterior muscles.

Myotonometer Assessment

Intervention Type DIAGNOSTIC_TEST

Muscle stiffness, muscle tone, relaxation periods and viscoelasticity will be assessed by a myotonometer of selected muscles (non-invasive measurement). The method of measurement consists of recording damped natural oscillation of soft biological tissue in the form of an acceleration signal and the subsequent simultaneous computation of the parameters of State of Tension, Biomechanical and Viscoelastic properties. Measurements will be performed on the trapezius, deltoid and supraspinatus muscles, the rectus femoris muscles, and the tibialis anterior muscles.

Vital signs

Intervention Type DIAGNOSTIC_TEST

Vital signs (blood pressure, heart rate, respiratory rate) will be measured before and after the six-Minute-Walk-Test (6MWT).

Borg Scale

Intervention Type DIAGNOSTIC_TEST

the Borg scale will be assessed, which is a self-reported questionnaire designed to subjectively assess dyspnea and exertion during activity (Borg 1982). The Borg scale rates dyspnea on a scale of 0 to 10 incorporating nonlinear spacing of verbal descriptors of the level of intensity of dyspnea. A higher Borg score indicates more severe dyspnea. The Borg scale will be administered before starting the 6MWT (≤ 5 minutes) and after completing the 6MWT (≤ 5 minutes).

Healthy control

no known neuromuscular disorder

Beck depression inventory fast screen

Intervention Type DIAGNOSTIC_TEST

Beck depression inventory fast screen questionnaire to detect severe depression for eligibility.

German Pain Inventory

Intervention Type DIAGNOSTIC_TEST

German Pain Inventory questionnaire for evaluation of pain. Module A, abbreviated questions of module S (sociodemographic questions S1, S2, S3, S4, S5 and S8) and module L (quality of life) and V (therapies) will be used.

Brief Pain Inventory

Intervention Type DIAGNOSTIC_TEST

Validated questionnaire for pain.

Fatigue Severity and Disability Scale (FSS)

Intervention Type DIAGNOSTIC_TEST

Validated questionnaire for perceived fatigue

Quick Motor Function Test

Intervention Type DIAGNOSTIC_TEST

An evaluator observes the performance of a patient and scores the items separately on a 5-point ordinal scale (ranging from 0 to 4). A total score is obtained by adding the scores of all items and ranges between 0 and 64 points.

Handheld Dynamometry (HHD)

Intervention Type DIAGNOSTIC_TEST

To ensure a high level of objective measurement, muscle strength will also be assessed by handheld dynamometry. The following muscle groups will be tested: Arm abduction, elbow flexion, elbow extension, hip flexion, hip extension, knee extension, knee flexion, foot extension, foot flexion.

Six-minute walk test (6MWT)

Intervention Type DIAGNOSTIC_TEST

It is a sub-maximal exercise test used to assess aerobic capacity and endurance. The distance covered over a time of 6 minutes is used as the outcome by which to compare changes in performance capacity.

Pressure pain threshold

Intervention Type DIAGNOSTIC_TEST

For diagnosis of myofascial pain, pressure algometers are designed and conventionally used to measure deep pressure pain thresholds or tenderness resistance (Park, Kim et al. 2011), and the reliability of pressure pain thresholds according to raters or measurement frequencies has been proven to be relatively high (Chung, Um et al. 1992). The threshold is then determined as the arithmetic mean of the 3 series (in kPa). The measurement will be stopped immediately as the patient feels sensations of "burning", "stinging", "drilling" or "aching. Pressure algometry measurements will be performed on the trapezius, deltoid and supraspinatus muscles, the rectus femoris muscles, and the tibialis anterior muscles.

Myotonometer Assessment

Intervention Type DIAGNOSTIC_TEST

Muscle stiffness, muscle tone, relaxation periods and viscoelasticity will be assessed by a myotonometer of selected muscles (non-invasive measurement). The method of measurement consists of recording damped natural oscillation of soft biological tissue in the form of an acceleration signal and the subsequent simultaneous computation of the parameters of State of Tension, Biomechanical and Viscoelastic properties. Measurements will be performed on the trapezius, deltoid and supraspinatus muscles, the rectus femoris muscles, and the tibialis anterior muscles.

Vital signs

Intervention Type DIAGNOSTIC_TEST

Vital signs (blood pressure, heart rate, respiratory rate) will be measured before and after the six-Minute-Walk-Test (6MWT).

Borg Scale

Intervention Type DIAGNOSTIC_TEST

the Borg scale will be assessed, which is a self-reported questionnaire designed to subjectively assess dyspnea and exertion during activity (Borg 1982). The Borg scale rates dyspnea on a scale of 0 to 10 incorporating nonlinear spacing of verbal descriptors of the level of intensity of dyspnea. A higher Borg score indicates more severe dyspnea. The Borg scale will be administered before starting the 6MWT (≤ 5 minutes) and after completing the 6MWT (≤ 5 minutes).

Interventions

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Beck depression inventory fast screen

Beck depression inventory fast screen questionnaire to detect severe depression for eligibility.

Intervention Type DIAGNOSTIC_TEST

German Pain Inventory

German Pain Inventory questionnaire for evaluation of pain. Module A, abbreviated questions of module S (sociodemographic questions S1, S2, S3, S4, S5 and S8) and module L (quality of life) and V (therapies) will be used.

Intervention Type DIAGNOSTIC_TEST

Brief Pain Inventory

Validated questionnaire for pain.

Intervention Type DIAGNOSTIC_TEST

Fatigue Severity and Disability Scale (FSS)

Validated questionnaire for perceived fatigue

Intervention Type DIAGNOSTIC_TEST

Quick Motor Function Test

An evaluator observes the performance of a patient and scores the items separately on a 5-point ordinal scale (ranging from 0 to 4). A total score is obtained by adding the scores of all items and ranges between 0 and 64 points.

Intervention Type DIAGNOSTIC_TEST

Handheld Dynamometry (HHD)

To ensure a high level of objective measurement, muscle strength will also be assessed by handheld dynamometry. The following muscle groups will be tested: Arm abduction, elbow flexion, elbow extension, hip flexion, hip extension, knee extension, knee flexion, foot extension, foot flexion.

Intervention Type DIAGNOSTIC_TEST

Six-minute walk test (6MWT)

It is a sub-maximal exercise test used to assess aerobic capacity and endurance. The distance covered over a time of 6 minutes is used as the outcome by which to compare changes in performance capacity.

Intervention Type DIAGNOSTIC_TEST

Pressure pain threshold

For diagnosis of myofascial pain, pressure algometers are designed and conventionally used to measure deep pressure pain thresholds or tenderness resistance (Park, Kim et al. 2011), and the reliability of pressure pain thresholds according to raters or measurement frequencies has been proven to be relatively high (Chung, Um et al. 1992). The threshold is then determined as the arithmetic mean of the 3 series (in kPa). The measurement will be stopped immediately as the patient feels sensations of "burning", "stinging", "drilling" or "aching. Pressure algometry measurements will be performed on the trapezius, deltoid and supraspinatus muscles, the rectus femoris muscles, and the tibialis anterior muscles.

Intervention Type DIAGNOSTIC_TEST

Myotonometer Assessment

Muscle stiffness, muscle tone, relaxation periods and viscoelasticity will be assessed by a myotonometer of selected muscles (non-invasive measurement). The method of measurement consists of recording damped natural oscillation of soft biological tissue in the form of an acceleration signal and the subsequent simultaneous computation of the parameters of State of Tension, Biomechanical and Viscoelastic properties. Measurements will be performed on the trapezius, deltoid and supraspinatus muscles, the rectus femoris muscles, and the tibialis anterior muscles.

Intervention Type DIAGNOSTIC_TEST

Vital signs

Vital signs (blood pressure, heart rate, respiratory rate) will be measured before and after the six-Minute-Walk-Test (6MWT).

Intervention Type DIAGNOSTIC_TEST

Borg Scale

the Borg scale will be assessed, which is a self-reported questionnaire designed to subjectively assess dyspnea and exertion during activity (Borg 1982). The Borg scale rates dyspnea on a scale of 0 to 10 incorporating nonlinear spacing of verbal descriptors of the level of intensity of dyspnea. A higher Borg score indicates more severe dyspnea. The Borg scale will be administered before starting the 6MWT (≤ 5 minutes) and after completing the 6MWT (≤ 5 minutes).

Intervention Type DIAGNOSTIC_TEST

Other Intervention Names

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BDI-FS Deutscher Schmerzfragebogen (DSF) BPI FSS QMFT HHD 6MWT PPT Vital parameters (VP)

Eligibility Criteria

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Inclusion Criteria

* The participant is willing and able to provide signed informed consent.
* The participant is able and willing to perform study-related assessments.
* The participant is ≥18 years of age
* The participant has one of the following diagnoses:

* histologically confirmed inclusion body myositis (IBM), or
* genetically confirmed late-onset Pompe disease (LOPD), or
* genetically confirmed spinal muscular atrophy type 3 (SMA3), or
* genetically confirmed myotonic dystrophy type 1, or
* genetically confirmed myotonic dystrophy type 2, or
* genetically confirmed facio-scapulo-humeral muscle dystrophy (FSHD).

Exclusion Criteria

* The participant is participating in another clinical study or using an investigational treatment.
* The participant, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
* The participant has currently a severe depression, assessed by the Beck depression inventory fast screen (BDI-FS) with a score ≥ 4
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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LMU Klinikum

OTHER

Sponsor Role lead

Responsible Party

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Prof. Dr. Benedikt Schoser

Clinical Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Stephan Wenninger, PD Dr. med.

Role: PRINCIPAL_INVESTIGATOR

Neurologist

Locations

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Friedrich-Baur-Institute

München, Bavaria, Germany

Site Status

Countries

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Germany

Other Identifiers

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PainNMD Version 1

Identifier Type: -

Identifier Source: org_study_id

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