Back to resultsDM-IMT - Controlled, Randomized, Three-arm Intervention Study on the Safety and Efficacy of Regular Respiratory Muscle Training in Patients With Myotonic Dystrophy Type 1 (DM-IMT)
Primary Purpose
Myotonic Dystrophy 1
Status
Completed
Phase
Not Applicable
Locations
Germany
Study Type
Interventional
Intervention
IMT - inspiratory muscle training
Sponsored by
About this trial
This is an interventional supportive care trial for Myotonic Dystrophy 1
Eligibility Criteria
Inclusion Criteria:
- the patient is willing and able to provide a signed informed consent form
- the patient is ≥ 18 years old
- the diagnosis of type 1 myotonic dystrophy has been confirmed by molecular genetics
- the patient is able and willing to perform pulmonary function tests (PFT) and blood sampling for capillary blood gas analysis (pO2, pCO2) throughout the study, to keep a diary and to complete questionnaires
Exclusion Criteria:
- the patient requires invasive ventilation (non-invasive ventilation is allowed).
- the patient uses non-invasive ventilation more than 16h/day.
- the patient participates in another clinical study that involves therapy.
- the patient cannot perform pulmonary function tests (PFT).
- the patient is diagnosed with central sleep apnea in polysomnography and not sufficiently treated with NIV ventilation.
- the patient is diagnosed with obstructive sleep apnea and not sufficiently treated with NIV ventilation.
- the patient cannot meet the requirements of the study, according to the investigator.
- the patient is unable to complete a 6-minute walking test
Sites / Locations
- Friedrich-Baur-Institute, Dep. of Neurology Klinikum der Universitaet Muenchen Munich, Germany
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Active Comparator
Active Comparator
No Intervention
Arm Label
Group 1: Strength Training
Group 2: Endurance Training
Group 3: Control group
Arm Description
Respiratory muscle strength training
Respiratory muscle endurance training
no training of respiratory muscles
Outcomes
Primary Outcome Measures
Safety of recurrent inspiratory muscle training
Intervention-related (serious) adverse events will be assessed as described in protocoll DM-IMT Version 1.2 (26/June/2019). For example a deterioration of > 15% of the FVC compared to the baseline measurements is defined as AE, as is the development of unusual myalgia of the respiratory muscles for more than 12 hours after the respiratory training. Detailed lists of patients experiencing adverse events or SAEs are reported. The severity of the adverse event is classified as mild, moderate or severe. Relationships of an AE to the training are categorized as unassociated, unlikely to be associated, possibly associated or associated. A separate list will be provided for patients who drop out of the study due to AEs. The frequency of adverse events leading to study termination is also summarized. Safety parameters also include lung function tests (PFT, including FVC, FEV1, MIP, MEP), physical examination, vital signs and clinical laboratory tests as required.
Secondary Outcome Measures
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by MIP.
The efficacy of the respiratory training is determined by the results of Maximal inspiratory pressure (MIP).
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by MIP.
The efficacy of the respiratory training is determined by the results of Maximal inspiratory pressure (MIP).
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by MEP.
The efficacy of the respiratory training is determined by the results of Maximal exspiratory pressure (MEP).
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by MEP.
The efficacy of the respiratory training is determined by the results of Maximal exspiratory pressure (MEP).
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by FVC.
The efficacy of the respiratory training is determined by the results of Forced Vital capacity (FVC).
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by FVC.
The efficacy of the respiratory training is determined by the results of Forced Vital capacity (FVC).
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by FEV1.
The efficacy of the respiratory training is determined by the results of Forced Expiratory Volume in 1 second (FEV1).
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by FEV1.
The efficacy of the respiratory training is determined by the results of Forced Expiratory Volume in 1 second (FEV1).
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by pCO2.
The efficacy of the respiratory training is determined by the results of pCO2 in capillary blood gas analysis.
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by pCO2.
The efficacy of the respiratory training is determined by the results of pCO2 in capillary blood gas analysis.
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by pO2.
The efficacy of the respiratory training is determined by the results of pO2 in capillary blood gas analysis.
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by pO2.
The efficacy of the respiratory training is determined by the results of pO2 in capillary blood gas analysis.
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by pH.
The efficacy of the respiratory training is determined by the results of pH in capillary blood gas analysis.
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by pH.
The efficacy of the respiratory training is determined by the results of pH in capillary blood gas analysis.
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by 6-minute-walk-test.
The efficacy of the respiratory training is determined by the results of 6-minute-walk-test.
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training compared to patients without training, measured by DM1-Activ.
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training will be measured by the result of DM1-ACTIV, a Rasch-built DM activity and participation scale for clinical use.
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training compared to patients without training, measured by FDSS.
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training will be measured by the result of the FDSS - FATIGUE AND DAYTIME SLEEPINESS SCALE, a Rasch-built combined fatigue and daytime sleepiness scale specifically designed for patients with DM1.
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training compared to patients without training, measured by the questionnaire Respicheck.
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training will be measured by the result of the RESPICHECK, a questionnaire on clinical symptoms of respiratory insufficiency.
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training compared to patients without training, measured by a physical examination.
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training will be measured by the result of the physical examination.
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training compared to patients without training, using the 6-minute-walk-test.
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training will be measured by the result of the 6-minute-walk-test.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04052958
Brief Title
DM-IMT - Controlled, Randomized, Three-arm Intervention Study on the Safety and Efficacy of Regular Respiratory Muscle Training in Patients With Myotonic Dystrophy Type 1
Acronym
DM-IMT
Official Title
DM-IMT - Controlled, Randomized, Three-arm Intervention Study on the Safety and Efficacy of Regular Respiratory Muscle Training in Patients With Myotonic Dystrophy Type 1
Study Type
Interventional
2. Study Status
Record Verification Date
April 2022
Overall Recruitment Status
Completed
Study Start Date
August 15, 2019 (Actual)
Primary Completion Date
December 31, 2021 (Actual)
Study Completion Date
December 31, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
LMU Klinikum
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
For some diseases, regular respiratory muscle training could delay the start of ventilation. For DM1, however, there are no clinically high-quality studies. Only a case description from the year 2006 showed a missing improvement of the symptoms after respiratory muscle training in one patient, accordingly there are no recommendations in this issue. Within the scope of this monocentric, three-arm, controlled intervention study, 45 patients with genetically confirmed type 1 myotonic dystrophy will be randomized in three groups of 15 patients each, age-, gender- and symptom-corrected by the MUSCULAR IMPAIRMENT RATING SCALE (MIRS). The DM1 patients will receive regular respiratory muscle training for a period of 9 months. The aim of this study is to evaluate the safety and effectiveness of regular inspiratory strength-breathing muscle training on 15 patients, the safety and effectiveness of regular inspiratory endurance respiratory muscle training on 15 patients, and the comparison to the natural course in 15 patients without training. Subsequently, we will provide treatment recommendations for respiratory training in DM1.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myotonic Dystrophy 1
7. Study Design
Primary Purpose
Supportive Care
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
45 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Group 1: Strength Training
Arm Type
Active Comparator
Arm Description
Respiratory muscle strength training
Arm Title
Group 2: Endurance Training
Arm Type
Active Comparator
Arm Description
Respiratory muscle endurance training
Arm Title
Group 3: Control group
Arm Type
No Intervention
Arm Description
no training of respiratory muscles
Intervention Type
Device
Intervention Name(s)
IMT - inspiratory muscle training
Intervention Description
respiratory strength or indurance training with respiratory therapy device
Primary Outcome Measure Information:
Title
Safety of recurrent inspiratory muscle training
Description
Intervention-related (serious) adverse events will be assessed as described in protocoll DM-IMT Version 1.2 (26/June/2019). For example a deterioration of > 15% of the FVC compared to the baseline measurements is defined as AE, as is the development of unusual myalgia of the respiratory muscles for more than 12 hours after the respiratory training. Detailed lists of patients experiencing adverse events or SAEs are reported. The severity of the adverse event is classified as mild, moderate or severe. Relationships of an AE to the training are categorized as unassociated, unlikely to be associated, possibly associated or associated. A separate list will be provided for patients who drop out of the study due to AEs. The frequency of adverse events leading to study termination is also summarized. Safety parameters also include lung function tests (PFT, including FVC, FEV1, MIP, MEP), physical examination, vital signs and clinical laboratory tests as required.
Time Frame
nine months
Secondary Outcome Measure Information:
Title
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by MIP.
Description
The efficacy of the respiratory training is determined by the results of Maximal inspiratory pressure (MIP).
Time Frame
nine months
Title
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by MIP.
Description
The efficacy of the respiratory training is determined by the results of Maximal inspiratory pressure (MIP).
Time Frame
nine months
Title
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by MEP.
Description
The efficacy of the respiratory training is determined by the results of Maximal exspiratory pressure (MEP).
Time Frame
nine months
Title
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by MEP.
Description
The efficacy of the respiratory training is determined by the results of Maximal exspiratory pressure (MEP).
Time Frame
nine months
Title
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by FVC.
Description
The efficacy of the respiratory training is determined by the results of Forced Vital capacity (FVC).
Time Frame
nine months
Title
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by FVC.
Description
The efficacy of the respiratory training is determined by the results of Forced Vital capacity (FVC).
Time Frame
nine months
Title
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by FEV1.
Description
The efficacy of the respiratory training is determined by the results of Forced Expiratory Volume in 1 second (FEV1).
Time Frame
nine months
Title
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by FEV1.
Description
The efficacy of the respiratory training is determined by the results of Forced Expiratory Volume in 1 second (FEV1).
Time Frame
nine months
Title
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by pCO2.
Description
The efficacy of the respiratory training is determined by the results of pCO2 in capillary blood gas analysis.
Time Frame
nine months
Title
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by pCO2.
Description
The efficacy of the respiratory training is determined by the results of pCO2 in capillary blood gas analysis.
Time Frame
nine months
Title
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by pO2.
Description
The efficacy of the respiratory training is determined by the results of pO2 in capillary blood gas analysis.
Time Frame
nine months
Title
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by pO2.
Description
The efficacy of the respiratory training is determined by the results of pO2 in capillary blood gas analysis.
Time Frame
nine months
Title
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by pH.
Description
The efficacy of the respiratory training is determined by the results of pH in capillary blood gas analysis.
Time Frame
nine months
Title
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by pH.
Description
The efficacy of the respiratory training is determined by the results of pH in capillary blood gas analysis.
Time Frame
nine months
Title
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by 6-minute-walk-test.
Description
The efficacy of the respiratory training is determined by the results of 6-minute-walk-test.
Time Frame
nine months
Title
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training compared to patients without training, measured by DM1-Activ.
Description
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training will be measured by the result of DM1-ACTIV, a Rasch-built DM activity and participation scale for clinical use.
Time Frame
nine months
Title
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training compared to patients without training, measured by FDSS.
Description
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training will be measured by the result of the FDSS - FATIGUE AND DAYTIME SLEEPINESS SCALE, a Rasch-built combined fatigue and daytime sleepiness scale specifically designed for patients with DM1.
Time Frame
nine months
Title
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training compared to patients without training, measured by the questionnaire Respicheck.
Description
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training will be measured by the result of the RESPICHECK, a questionnaire on clinical symptoms of respiratory insufficiency.
Time Frame
nine months
Title
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training compared to patients without training, measured by a physical examination.
Description
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training will be measured by the result of the physical examination.
Time Frame
nine months
Title
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training compared to patients without training, using the 6-minute-walk-test.
Description
Impact on the quality of life and muscular performance in DM1 patients after recurrent respiratory muscle training will be measured by the result of the 6-minute-walk-test.
Time Frame
nine months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
the patient is willing and able to provide a signed informed consent form
the patient is ≥ 18 years old
the diagnosis of type 1 myotonic dystrophy has been confirmed by molecular genetics
the patient is able and willing to perform pulmonary function tests (PFT) and blood sampling for capillary blood gas analysis (pO2, pCO2) throughout the study, to keep a diary and to complete questionnaires
Exclusion Criteria:
the patient requires invasive ventilation (non-invasive ventilation is allowed).
the patient uses non-invasive ventilation more than 16h/day.
the patient participates in another clinical study that involves therapy.
the patient cannot perform pulmonary function tests (PFT).
the patient is diagnosed with central sleep apnea in polysomnography and not sufficiently treated with NIV ventilation.
the patient is diagnosed with obstructive sleep apnea and not sufficiently treated with NIV ventilation.
the patient cannot meet the requirements of the study, according to the investigator.
the patient is unable to complete a 6-minute walking test
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Stephan Wenninger, Dr.med.
Organizational Affiliation
Neurologist
Official's Role
Principal Investigator
Facility Information:
Facility Name
Friedrich-Baur-Institute, Dep. of Neurology Klinikum der Universitaet Muenchen Munich, Germany
City
Munich
State/Province
Bavaria
ZIP/Postal Code
80336
Country
Germany
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
DM-IMT - Controlled, Randomized, Three-arm Intervention Study on the Safety and Efficacy of Regular Respiratory Muscle Training in Patients With Myotonic Dystrophy Type 1
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