Pompe Disease (Late-onset)NCT04907162
Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders
An observational study of Pompe Disease (Late-onset) and Myotonic Dystrophy Type 1 (DM1), run by LMU Klinikum at 1 site in Germany.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Pompe Disease (Late-onset), Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy Type 2
- What is being tested?
- Beck depression inventory fast screen, German Pain Inventory and Brief Pain Inventory
- How many people
- 82 participants
- Where
- 1 siteGermany
- Study dates
- 15 Apr 2021to 15 Aug 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
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.
Who may be able to join
- 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
Show all 10Show fewer
- 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).
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Nociceptive Pain in Neuromuscular Disorders - Low Interventional Pilot Study to Assess Musculoskeletal Pain in Neuromuscular Disorders (NMD)
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.
Read the 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.
- Conditions
- 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
- Being tested
- Beck depression inventory fast screen, German Pain Inventory, Brief Pain Inventory, Fatigue Severity and Disability Scale (FSS), Quick Motor Function Test
- Keywords
- nociceptive pain, musculoskeletal
- Medical subject headings
- Glycogen Storage Disease Type II, Myotonic Dystrophy, Spinal Muscular Atrophies of Childhood, Myositis, Inclusion Body, Nociceptive Pain
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Beck depression inventory fast screenDiagnostic test
Also called BDI-FS
Beck depression inventory fast screen questionnaire to detect severe depression for eligibility.
German Pain InventoryDiagnostic test
Also called Deutscher Schmerzfragebogen (DSF)
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 InventoryDiagnostic test
Also called BPI
Validated questionnaire for pain.
Fatigue Severity and Disability Scale (FSS)Diagnostic test
Also called FSS
Validated questionnaire for perceived fatigue
Quick Motor Function TestDiagnostic test
Also called QMFT
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)Diagnostic test
Also called 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.
Six-minute walk test (6MWT)Diagnostic test
Also called 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.
Pressure pain thresholdDiagnostic test
Also called PPT
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 AssessmentDiagnostic 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 signsDiagnostic test
Also called Vital parameters (VP)
Vital signs (blood pressure, heart rate, respiratory rate) will be measured before and after the six-Minute-Walk-Test (6MWT).
Borg ScaleDiagnostic 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).
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: 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 | |
| Healthy control | no known neuromuscular disorder Receives: 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 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Prevalence of musculoskeletal pain in defined neuromuscular diseases
The primary aim is to characterize the prevalence of musculoskeletal pain in adult patients with neuromuscular disorders (NMD).
Measured: Only at baseline visit
Secondary outcomes (9)
Association between musculoskeletal pain and muscle function, assessed by Medical research council (MRC) grading (0-5)
Assessed by Medical research council (MRC) grading (0-5)
Measured: Only at baseline visit
Association between musculoskeletal pain and muscle function, assessed by quick motor function test (QMFT)
Assessed by quick motor function test (QMFT)
Measured: Only at baseline visit
Association between musculoskeletal pain and muscle function, assessed by Pressure Pain Threshold (PPT)
Assessed by Pressure Pain Threshold (PPT)
Measured: Only at baseline visit
Association between musculoskeletal pain and muscle function, assessed by a Myotonometer
Assessed by a Myotonometer (incl. relaxation time, stiffness, muscle tone, relaxation periods and viscoelasticity)
Measured: Only at baseline visit
Assessment of Questionnaire: Beck depression inventory fast screen
Association between Depression and musculoskeletal pain
Measured: Only at baseline visit
Assessment of Questionnaire: Brief Pain Inventory
Association between Depression and musculoskeletal pain
Measured: Only at baseline visit
Assessment of Questionnaire: Fatigue Severity Scale (FSS)
Association between Depression and musculoskeletal pain
Measured: Only at baseline visit
Characterization of musculoskeletal pain (quality and severity) assessed by the German Pain Questionnaire
Characterization of quality and severity of musculoskeletal pain
Measured: Only at baseline visit
Characterization of musculoskeletal pain (quality and severity) assessed by the Brief Pain Inventory (BPI)
Characterization (quality and severity) of musculoskeletal pain
Measured: Only at baseline visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2021
- Study start15 Apr 2021
- Primary completion15 Aug 2022
- Study completion15 Aug 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe Disease (Late-onset)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 82 participants (actual)
Sponsor and investigators
- Lead sponsor
- LMU Klinikum
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Prof. Dr. Benedikt Schoser, LMU Klinikum
- Investigators
- Stephan Wenninger, PD Dr. med., Principal investigator, Neurologist
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04907162
- Sponsor's protocol ID
- PainNMD Version 1
- Status last verified
- Aug 2022
- First submitted
- 21 May 2021
- Last updated
- 24 Aug 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Nociceptive Pain in Neuromuscular Disorders - Low Interventional Pilot Study to Assess Musculoskeletal Pain in Neuromuscular Disorders (NMD)
- ClinicalTrials.gov ID
- NCT04907162
- Acronym / sponsor ID
- PainNMD Version 1
- Conditions
- 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
- Keywords
- nociceptive pain, musculoskeletal
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 82 participants (actual)
- Lead sponsor
- LMU KlinikumUniversity, hospital or other
- Study start
- 15 Apr 2021 (actual)
- Primary completion
- 15 Aug 2022 (actual)
- Study completion
- 15 Aug 2022 (actual)
- First posted
- 28 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Neuromuscular disease (NMD) Patients | – | 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 ScaleThe 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). |
| Healthy control | – | 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 Scaleno known neuromuscular disorder |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (10 listed)
- 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:
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Beck depression inventory fast screenAlso: BDI-FSBeck depression inventory fast screen questionnaire to detect severe depression for eligibility. | Diagnostic test | Healthy control, Neuromuscular disease (NMD) Patients |
| German Pain InventoryAlso: Deutscher Schmerzfragebogen (DSF)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. | Diagnostic test | Healthy control, Neuromuscular disease (NMD) Patients |
| Brief Pain InventoryAlso: BPIValidated questionnaire for pain. | Diagnostic test | Healthy control, Neuromuscular disease (NMD) Patients |
| Fatigue Severity and Disability Scale (FSS)Also: FSSValidated questionnaire for perceived fatigue | Diagnostic test | Healthy control, Neuromuscular disease (NMD) Patients |
| Quick Motor Function TestAlso: QMFTAn 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. | Diagnostic test | Healthy control, Neuromuscular disease (NMD) Patients |
| Handheld Dynamometry (HHD)Also: HHDTo 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. | Diagnostic test | Healthy control, Neuromuscular disease (NMD) Patients |
| Six-minute walk test (6MWT)Also: 6MWTIt 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. | Diagnostic test | Healthy control, Neuromuscular disease (NMD) Patients |
| Pressure pain thresholdAlso: PPTFor 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. | Diagnostic test | Healthy control, Neuromuscular disease (NMD) Patients |
| Myotonometer AssessmentMuscle 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 | Healthy control, Neuromuscular disease (NMD) Patients |
| Vital signsAlso: Vital parameters (VP)Vital signs (blood pressure, heart rate, respiratory rate) will be measured before and after the six-Minute-Walk-Test (6MWT). | Diagnostic test | Healthy control, Neuromuscular disease (NMD) Patients |
| Borg Scalethe 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). | Diagnostic test | Healthy control, Neuromuscular disease (NMD) Patients |
Primary outcome
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).
9 secondary outcomes
Association between musculoskeletal pain and muscle function, assessed by Medical research council (MRC) grading (0-5)
Time frame: Only at baseline visit
Association between musculoskeletal pain and muscle function, assessed by quick motor function test (QMFT)
Time frame: Only at baseline visit
Association between musculoskeletal pain and muscle function, assessed by Pressure Pain Threshold (PPT)
Time frame: Only at baseline visit
Association between musculoskeletal pain and muscle function, assessed by a Myotonometer
Time frame: Only at baseline visit
Assessment of Questionnaire: Beck depression inventory fast screen
Time frame: Only at baseline visit
Assessment of Questionnaire: Brief Pain Inventory
Time frame: Only at baseline visit
Assessment of Questionnaire: Fatigue Severity Scale (FSS)
Time frame: Only at baseline visit
Characterization of musculoskeletal pain (quality and severity) assessed by the German Pain Questionnaire
Time frame: Only at baseline visit
Characterization of musculoskeletal pain (quality and severity) assessed by the Brief Pain Inventory (BPI)
Time frame: Only at baseline visit
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Stephan Wenninger, PD Dr. med. principal investigator
Neurologist
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pompe Disease (Late-onset) and Myotonic Dystrophy Type 1 (DM1), run by LMU Klinikum at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Prevalence of musculoskeletal pain in defined neuromuscular diseases (time frame: Only at baseline visit). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Aug 2022.