Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04907162
Recruitment Status : Recruiting
First Posted : May 28, 2021
Last Update Posted : June 11, 2021
Sponsor:
Information provided by (Responsible Party):
Prof. Dr. Benedikt Schoser, LMU Klinikum

Brief Summary:
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.

Condition or disease Intervention/treatment
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 Diagnostic Test: Beck depression inventory fast screen Diagnostic Test: German Pain Inventory Diagnostic Test: Brief Pain Inventory Diagnostic Test: Fatigue Severity and Disability Scale (FSS) Diagnostic Test: Quick Motor Function Test Diagnostic Test: Handheld Dynamometry (HHD) Diagnostic Test: Six-minute walk test (6MWT) Diagnostic Test: Pressure pain threshold Diagnostic Test: Myotonometer Assessment Diagnostic Test: Vital signs Diagnostic Test: Borg Scale

Detailed Description:
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.

Layout table for study information
Study Type : Observational
Estimated Enrollment : 90 participants
Observational Model: Case-Control
Time Perspective: Prospective
Official Title: Nociceptive Pain in Neuromuscular Disorders - Low Interventional Pilot Study to Assess Musculoskeletal Pain in Neuromuscular Disorders (NMD)
Actual Study Start Date : April 15, 2021
Estimated Primary Completion Date : June 2022
Estimated Study Completion Date : August 2022


Group/Cohort Intervention/treatment
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).
Diagnostic Test: Beck depression inventory fast screen
Beck depression inventory fast screen questionnaire to detect severe depression for eligibility.
Other Name: BDI-FS

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.
Other Name: Deutscher Schmerzfragebogen (DSF)

Diagnostic Test: Brief Pain Inventory
Validated questionnaire for pain.
Other Name: BPI

Diagnostic Test: Fatigue Severity and Disability Scale (FSS)
Validated questionnaire for perceived fatigue
Other Name: FSS

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.
Other Name: QMFT

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.
Other Name: HHD

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.
Other Name: 6MWT

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.
Other Name: PPT

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.

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

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).

Healthy control
no known neuromuscular disorder
Diagnostic Test: Beck depression inventory fast screen
Beck depression inventory fast screen questionnaire to detect severe depression for eligibility.
Other Name: BDI-FS

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.
Other Name: Deutscher Schmerzfragebogen (DSF)

Diagnostic Test: Brief Pain Inventory
Validated questionnaire for pain.
Other Name: BPI

Diagnostic Test: Fatigue Severity and Disability Scale (FSS)
Validated questionnaire for perceived fatigue
Other Name: FSS

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.
Other Name: QMFT

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.
Other Name: HHD

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.
Other Name: 6MWT

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.
Other Name: PPT

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.

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

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).




Primary Outcome Measures :
  1. Prevalence of musculoskeletal pain in defined neuromuscular diseases [ Time Frame: Only at baseline visit ]
    The primary aim is to characterize the prevalence of musculoskeletal pain in adult patients with neuromuscular disorders (NMD).


Secondary Outcome Measures :
  1. Association between musculoskeletal pain and muscle function, assessed by Medical research council (MRC) grading (0-5) [ Time Frame: Only at baseline visit ]
    Assessed by Medical research council (MRC) grading (0-5)

  2. Association between musculoskeletal pain and muscle function, assessed by quick motor function test (QMFT) [ Time Frame: Only at baseline visit ]
    Assessed by quick motor function test (QMFT)

  3. Association between musculoskeletal pain and muscle function, assessed by Pressure Pain Threshold (PPT) [ Time Frame: Only at baseline visit ]
    Assessed by Pressure Pain Threshold (PPT)

  4. Association between musculoskeletal pain and muscle function, assessed by a Myotonometer [ Time Frame: Only at baseline visit ]
    Assessed by a Myotonometer (incl. relaxation time, stiffness, muscle tone, relaxation periods and viscoelasticity)

  5. Assessment of Questionnaire: Beck depression inventory fast screen [ Time Frame: Only at baseline visit ]
    Association between Depression and musculoskeletal pain

  6. Assessment of Questionnaire: Brief Pain Inventory [ Time Frame: Only at baseline visit ]
    Association between Depression and musculoskeletal pain

  7. Assessment of Questionnaire: Fatigue Severity Scale (FSS) [ Time Frame: Only at baseline visit ]
    Association between Depression and musculoskeletal pain

  8. Characterization of musculoskeletal pain (quality and severity) assessed by the German Pain Questionnaire [ Time Frame: Only at baseline visit ]
    Characterization of quality and severity of musculoskeletal pain

  9. Characterization of musculoskeletal pain (quality and severity) assessed by the Brief Pain Inventory (BPI) [ Time Frame: Only at baseline visit ]
    Characterization (quality and severity) of musculoskeletal pain



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Probability Sample
Study Population
Approximately 70 patients with defined NMD (see inclusion criteria) will be enrolled. For a control group, 20 healthy volunteers will be enrolled. All patients must be ≥ 18 years of age.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04907162


Contacts
Layout table for location contacts
Contact: Stephan Wenninger, PD Dr. med. 49089440057470 stephan.wenninger@med.uni-muenchen.de
Contact: Elena Sagerer 49089440057470 s-esagerer@helios.med.uni-muenchen.de

Locations
Layout table for location information
Germany
Friedrich-Baur-Institute Recruiting
München, Bavaria, Germany, 80336
Principal Investigator: Stephan Wenninger, PD Dr. med.         
Sponsors and Collaborators
LMU Klinikum
Investigators
Layout table for investigator information
Principal Investigator: Stephan Wenninger, PD Dr. med. Neurologist
Layout table for additonal information
Responsible Party: Prof. Dr. Benedikt Schoser, Clinical Professor, LMU Klinikum
ClinicalTrials.gov Identifier: NCT04907162    
Other Study ID Numbers: PainNMD Version 1
First Posted: May 28, 2021    Key Record Dates
Last Update Posted: June 11, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Prof. Dr. Benedikt Schoser, LMU Klinikum:
nociceptive pain
musculoskeletal
Additional relevant MeSH terms:
Layout table for MeSH terms
Muscular Dystrophies
Myositis
Myotonic Dystrophy
Myositis, Inclusion Body
Muscular Dystrophy, Facioscapulohumeral
Muscular Atrophy
Muscular Atrophy, Spinal
Glycogen Storage Disease Type II
Neuromuscular Diseases
Nociceptive Pain
Atrophy
Pathological Conditions, Anatomical
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Neuromuscular Manifestations
Neurologic Manifestations
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Metabolism, Inborn Errors
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors