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Trial record 10 of 52 for:    Myotonic Dystrophy

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 (DM-IMT)

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ClinicalTrials.gov Identifier: NCT04052958
Recruitment Status : Not yet recruiting
First Posted : August 12, 2019
Last Update Posted : August 12, 2019
Sponsor:
Information provided by (Responsible Party):
Prof. Dr. Benedikt Schoser, Klinikum der Universitaet Muenchen

Tracking Information
First Submitted Date  ICMJE July 31, 2019
First Posted Date  ICMJE August 12, 2019
Last Update Posted Date August 12, 2019
Estimated Study Start Date  ICMJE August 15, 2019
Estimated Primary Completion Date December 15, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 9, 2019)
Safety of recurrent inspiratory muscle training [ Time Frame: nine months ]
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: August 9, 2019)
  • Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by MIP. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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. [ Time Frame: nine months ]
    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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Supportive Care
Condition  ICMJE Myotonic Dystrophy 1
Intervention  ICMJE Device: IMT - inspiratory muscle training
respiratory strength or indurance training with respiratory therapy device
Study Arms  ICMJE
  • Active Comparator: Group 1: Strength Training
    Respiratory muscle strength training
    Intervention: Device: IMT - inspiratory muscle training
  • Active Comparator: Group 2: Endurance Training
    Respiratory muscle endurance training
    Intervention: Device: IMT - inspiratory muscle training
  • No Intervention: Group 3: Control group
    no training of respiratory muscles
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: August 9, 2019)
45
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 15, 2021
Estimated Primary Completion Date December 15, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Stephan Wenninger, Dr.med. +49 89440057470 stephan.wenninger@med.uni-muenchen.de
Contact: Benedikt Schoser, Prof.Dr. +49 89440057470 benedikt.schoser@med.uni-muenchen.de
Listed Location Countries  ICMJE Germany
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04052958
Other Study ID Numbers  ICMJE Version 1.2 (26. Juni 2019)
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Prof. Dr. Benedikt Schoser, Klinikum der Universitaet Muenchen
Study Sponsor  ICMJE Klinikum der Universitaet Muenchen
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Stephan Wenninger, Dr.med. Neurologist
PRS Account Klinikum der Universitaet Muenchen
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP