Neuromuscular DisordersNCT04411732
Myotonia and Muscle Stiffness in NMD
An observational study of Neuromuscular Disorders, run by LMU Klinikum at 1 site in Germany.
Trial summary
The primary objective of this study is to assess stiffness, muscle tone, relaxation periods and elasticity of various muscles in patients with dystrophic or non-dystrophic myotonia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neuromuscular Disorders
- What is being tested?
- MyotonPro, Six-minute walk test and Semiquantitative muscle ultrasound
- Type of treatment
- Injection or infusion, Medical device
- How many people
- 133 participants
- Where
- 1 siteGermany
- Study dates
- 1 Oct 2019to 30 Sep 2023
- 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
- Adult, Older adult
Study population: Approximately 70 patients will be enrolled, about 3-5 each in the following disease groups: Limb-Girdle-Muscular Dystrophy (LGMD), Myotonic Dystrophies Type 1 and 2 (DM1, DM2), Late-Onset-Pompe Disease (LOPD), Distal Myopathies, Congenital Myotonia, Spinal muscular atrophy Type 3 (SMA3), Amyotrophic lateral sclerosis (ALS) and peripheral Polyneuropathies (CIDP, HMSN). 20 healthy age- and gender-matched patients will be enrolled as a control group.
Who may be able to join
- For Patients:
- ≥18 years of age
- Confirmed neuromuscular disease
- written consent
- able and willing to perform study prodcedures
- For healthy volunteers:
Show all 9Show fewer
- age ≥18 years of age
- written informed consent
- no clinical sign for neuromuscular disorders
Who cannot join
- Severe comorbidities
- Patient is participating in another clinical study using investigational treatment
- Patient cannot perform required muscle function tests
- the patient, in the opinion of the investigator, is unable to adhere to the requirements of the study
Trial locations and contacts
1 site in 1 country.
Germany1 site
Friedrich-Baur-Institute, Dep. of Neurology Klinikum der Universitaet Muenchen
Munich, Bavaria, 80336
About this study
Official title: Myotonia, Muscle Stiffness and Elasticity in Neuromuscular Disorders
The primary objective of this study is to assess stiffness, muscle tone, relaxation periods and elasticity of various muscles in patients with dystrophic or non-dystrophic myotonia. The secondary objectives are (1) to provide reference values for stiffness, muscle tone, relaxation periods and elasticity of various muscles in patients with dystrophic or non-dystrophic myotonia; to provide reference values for stiffness, muscle tone, relaxation periods and elasticity of various muscles in patients with non-myotonic neuromuscular disorders, (3) assess correlations between to compare result values for stiffness, muscle tone, relaxation periods and elasticity with clinical muscle function tests, measured by clinical evaluation (MRC-scale) and the 6-minute walk test; (4) assess correlations between subcutaneous fat and muscle thickness and echogenicity, measured by muscle ultrasound and result values for stiffness, muscle tone, relaxation periods and elasticity.
- Conditions
- Neuromuscular Disorders
- Being tested
- MyotonPro, Six-minute walk test, Semiquantitative muscle ultrasound, Hand-held Dynamometry of muscle strength
- Medical subject headings
- Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MyotonProDevice
Measurement of relaxation time, stiffness and elasticity of the following muscles, using the MyotonPro® device: on both sides thenar and hypothenar, m. biceps brachii, m. triceps brachii, m. deltoideus, m. quadriceps femoris, m. tibialis anterior and gastrocnemius muscles.
Six-minute walk testDiagnostic test
The six-minute walk test (6MWT) measures the distance an individual is able to walk over a total of six minutes on a hard, flat surface. The goal is for the individual to walk as far as possible in six minutes. The individual is allowed to self-pace and rest as needed as they traverse back and forth along a marked walkway. The 6MWT has been used with a variety of other conditions than the chronic obstructive pulmonary disease (COPD) such as heart failure and stroke and is widely used in neuromuscular diseases. The six-minute-walk-test will be conducted as recommended by the American Thoracic Society. In this study, the six-minute-walk test will be performed once on visit 1 to detect the impact of muscle weakness, muscle stiffness and myotonia on muscular endurance.
Semiquantitative muscle ultrasoundDiagnostic test
Muscle ultrasound is an ideal imaging modality that allows for atraumatic, noninvasive, radiation-free point-of-care neuromuscular imaging. Muscular dystrophies are typically associated with an increase in the echogenicity from the muscle substance, distal attenuation of muscle echo and a corresponding loss of bone echo. Spinal muscular atrophies and neuropathies also showed an increase in muscle echo along with atrophy of the muscle and increase in depth of subcutaneous tissue, but a persisting bone echo. In several other myopathies, similar changes are seen. In semi-quantitative muscle ultrasound, muscle intensity will be documented using the 4-point Heckmatt score. In addition to that, cutis, subcutaneus fat and muscle will be measured in mm.
Hand-held Dynamometry of muscle strengthDiagnostic test
Muscle strength will be assessed by handheld dynamometry using the MicroFET2 myometer, produced by Hoggan Health Industries. This test is widely used in patients with neuromuscular diseases. To perform a test, the examiner holds the dynamometer stationary while the patient exerts a maximal force against the dynamometer. The patient makes a gradual increase in force and then completes an isometric hold for 4-5 seconds. The following muscle groups will be tested: Arm abduction, elbow flexion, elbow extension, knee extension, knee flexion, foot extension, foot flexion.
| Study group | Role | What they receive |
|---|---|---|
| patients with neuromuscular disorder | cohort of patients with neuromuscular disorder Receives: Device: MyotonPro; Diagnostic Test: Six-minute walk test; Diagnostic Test: Semiquantitative muscle ultrasound; Diagnostic Test: Hand-held Dynamometry of muscle strength | |
| healthy controls | cohort of healthy controls Receives: Device: MyotonPro; Diagnostic Test: Six-minute walk test; Diagnostic Test: Semiquantitative muscle ultrasound; Diagnostic Test: Hand-held Dynamometry of muscle strength |
What is being measured
The primary outcome is the main question the study is designed to answer.
MyotonPro device in patients with dystrophic and non-dystrophic myotonia
The primary objective of this study is the comparison of viscoelastic stiffness of the underlying tissue measured by stiffness (S), elasticity (D) and relaxation (R) between patients with dystrophic and non-dystrophic myotonia.
Measured: 1 day
Secondary outcomes (5)
MyotonPro device - reference values in patients with dystrophic and non-dystrophic myotonia
The secondary objective of this study is to provide reference values for stiffness, muscle tone, relaxation periods and elasticity of various muscles in patients with dystrophic or non-dystrophic myotonia.
Measured: 1 day
MyotonPro device - reference values in patients with non-myotonic neuromuscular disorders
The secondary objective of this study is to provide reference values for stiffness, muscle tone, relaxation periods and elasticity of various muscles in patients with non-myotonic neuromuscular disorders.
Measured: 1 day
MyotonPro device - correlations between stiffness, muscle tone, relaxation periods and elasticity with clinical muscle function tests
The secondary objective of this study is to compare stiffness, muscle tone, relaxation periods and elasticity with clinical muscle function tests, measured by clinical evaluation (MRC-scale).
Measured: 1 day
MyotonPro device - correlations between stiffness, muscle tone, relaxation periods and elasticity with subcutaneous fat and muscle thickness and echogenicity
The secondary objective of this study is to assess correlations between subcutaneous fat and muscle thickness and echogenicity, measured by muscle ultrasound and result values for stiffness, muscle tone, relaxation periods and elasticity.
Measured: 1 day
MyotonPro device - correlations between stiffness, muscle tone, relaxation periods and elasticity and the 6-minute-walk test
The secondary objective of this study is to compare stiffness, muscle tone, relaxation periods and elasticity with clinical muscle endurance function tests, measured by the 6-minute walk test.
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jun 2020
- Study start1 Oct 2019
- Primary completion31 Jul 2023
- Study completion30 Sep 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular DisordersStudies 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
- 133 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, Dr. med., Principal investigator, Neurologist
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04411732
- Sponsor's protocol ID
- Version: 1 [01-JUL-2019]
- Status last verified
- Oct 2023
- First submitted
- 25 May 2020
- Last updated
- 11 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Myotonia, Muscle Stiffness and Elasticity in Neuromuscular Disorders
- ClinicalTrials.gov ID
- NCT04411732
- Acronym / sponsor ID
- Version: 1 [01-JUL-2019]
- Conditions
- Neuromuscular Disorders
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 133 participants (actual)
- Lead sponsor
- LMU KlinikumUniversity, hospital or other
- Study start
- 1 Oct 2019 (actual)
- Primary completion
- 31 Jul 2023 (actual)
- Study completion
- 30 Sep 2023 (actual)
- First posted
- 2 Jun 2020 (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 objective of this study is to assess stiffness, muscle tone, relaxation periods and elasticity of various muscles in patients with dystrophic or non-dystrophic myotonia. The secondary objectives are (1) to provide reference values for stiffness, muscle tone, relaxation periods and elasticity of various muscles in patients with dystrophic or non-dystrophic myotonia; to provide reference values for stiffness, muscle tone, relaxation periods and elasticity of various muscles in patients with non-myotonic neuromuscular disorders, (3) assess correlations between to compare result values for stiffness, muscle tone, relaxation periods and elasticity with clinical muscle function tests, measured by clinical evaluation (MRC-scale) and the 6-minute walk test; (4) assess correlations between subcutaneous fat and muscle thickness and echogenicity, measured by muscle ultrasound and result values for stiffness, muscle tone, relaxation periods and elasticity.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| patients with neuromuscular disorder | – | Device: MyotonPro, Diagnostic Test: Six-minute walk test, Diagnostic Test: Semiquantitative muscle ultrasound, Diagnostic Test: Hand-held Dynamometry of muscle strengthcohort of patients with neuromuscular disorder |
| healthy controls | – | Device: MyotonPro, Diagnostic Test: Six-minute walk test, Diagnostic Test: Semiquantitative muscle ultrasound, Diagnostic Test: Hand-held Dynamometry of muscle strengthcohort of healthy controls |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- Approximately 70 patients will be enrolled, about 3-5 each in the following disease groups: Limb-Girdle-Muscular Dystrophy (LGMD), Myotonic Dystrophies Type 1 and 2 (DM1, DM2), Late-Onset-Pompe Disease (LOPD), Distal Myopathies, Congenital Myotonia, Spinal muscular atrophy Type 3 (SMA3), Amyotrophic lateral sclerosis (ALS) and peripheral Polyneuropathies (CIDP, HMSN). 20 healthy age- and gender-matched patients will be enrolled as a control group.
Key inclusion criteria (9 listed)
- ≥18 years of age
- Confirmed neuromuscular disease
- written consent
- able and willing to perform study prodcedures
- age ≥18 years of age
Key exclusion criteria (4 listed)
- Severe comorbidities
- Patient is participating in another clinical study using investigational treatment
- Patient cannot perform required muscle function tests
- the patient, in the opinion of the investigator, is unable to adhere to the requirements of the study
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 |
|---|---|---|
| MyotonProMeasurement of relaxation time, stiffness and elasticity of the following muscles, using the MyotonPro® device: on both sides thenar and hypothenar, m. biceps brachii, m. triceps brachii, m. deltoideus, m. quadriceps femoris, m. tibialis anterior and gastrocnemius muscles. | Device | healthy controls, patients with neuromuscular disorder |
| Six-minute walk testThe six-minute walk test (6MWT) measures the distance an individual is able to walk over a total of six minutes on a hard, flat surface. The goal is for the individual to walk as far as possible in six minutes. The individual is allowed to self-pace and rest as needed as they traverse back and forth along a marked walkway. The 6MWT has been used with a variety of other conditions than the chronic obstructive pulmonary disease (COPD) such as heart failure and stroke and is widely used in neuromuscular diseases. The six-minute-walk-test will be conducted as recommended by the American Thoracic Society. In this study, the six-minute-walk test will be performed once on visit 1 to detect the impact of muscle weakness, muscle stiffness and myotonia on muscular endurance. | Diagnostic test | healthy controls, patients with neuromuscular disorder |
| Semiquantitative muscle ultrasoundMuscle ultrasound is an ideal imaging modality that allows for atraumatic, noninvasive, radiation-free point-of-care neuromuscular imaging. Muscular dystrophies are typically associated with an increase in the echogenicity from the muscle substance, distal attenuation of muscle echo and a corresponding loss of bone echo. Spinal muscular atrophies and neuropathies also showed an increase in muscle echo along with atrophy of the muscle and increase in depth of subcutaneous tissue, but a persisting bone echo. In several other myopathies, similar changes are seen. In semi-quantitative muscle ultrasound, muscle intensity will be documented using the 4-point Heckmatt score. In addition to that, cutis, subcutaneus fat and muscle will be measured in mm. | Diagnostic test | healthy controls, patients with neuromuscular disorder |
| Hand-held Dynamometry of muscle strengthMuscle strength will be assessed by handheld dynamometry using the MicroFET2 myometer, produced by Hoggan Health Industries. This test is widely used in patients with neuromuscular diseases. To perform a test, the examiner holds the dynamometer stationary while the patient exerts a maximal force against the dynamometer. The patient makes a gradual increase in force and then completes an isometric hold for 4-5 seconds. The following muscle groups will be tested: Arm abduction, elbow flexion, elbow extension, knee extension, knee flexion, foot extension, foot flexion. | Diagnostic test | healthy controls, patients with neuromuscular disorder |
Primary outcome
MyotonPro device in patients with dystrophic and non-dystrophic myotonia
Time frame: 1 day
The primary objective of this study is the comparison of viscoelastic stiffness of the underlying tissue measured by stiffness (S), elasticity (D) and relaxation (R) between patients with dystrophic and non-dystrophic myotonia.
5 secondary outcomes
MyotonPro device - reference values in patients with dystrophic and non-dystrophic myotonia
Time frame: 1 day
MyotonPro device - reference values in patients with non-myotonic neuromuscular disorders
Time frame: 1 day
MyotonPro device - correlations between stiffness, muscle tone, relaxation periods and elasticity with clinical muscle function tests
Time frame: 1 day
MyotonPro device - correlations between stiffness, muscle tone, relaxation periods and elasticity with subcutaneous fat and muscle thickness and echogenicity
Time frame: 1 day
MyotonPro device - correlations between stiffness, muscle tone, relaxation periods and elasticity and the 6-minute-walk test
Time frame: 1 day
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 11 Oct 2023.
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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, 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 Neuromuscular Disorders, 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 9 inclusion and 4 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 11 Oct 2023. 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?
MyotonPro device in patients with dystrophic and non-dystrophic myotonia (time frame: 1 day). 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 11 Oct 2023.