Clinical trial NCT04052958: 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: 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
Overall status: COMPLETED
Phase: NA
Conditions: Myotonic Dystrophy 1
Lead sponsor: LMU Klinikum
Sponsor class: OTHER
First posted: 2019-08-12
Enrollment: 45
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

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.


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

Primary outcomes:
measure: Safety of recurrent inspiratory muscle training
timeFrame: nine months
Secondary outcomes:
measure: Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by MIP.
timeFrame: nine months
measure: Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by MIP.
timeFrame: nine months
measure: Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by MEP.
timeFrame: nine months
measure: Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by MEP.
timeFrame: nine months
measure: Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by FVC.
timeFrame: nine months
measure: Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by FVC.
timeFrame: nine months
measure: Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by FEV1.
timeFrame: nine months
measure: Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by FEV1.
timeFrame: nine months
measure: Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by pCO2.
timeFrame: nine months
measure: Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by pCO2.
timeFrame: nine months
measure: Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by pO2.
timeFrame: nine months
measure: Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by pO2.
timeFrame: nine months
measure: Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by pH.
timeFrame: nine months
measure: Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by pH.
timeFrame: nine months
measure: Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by 6-minute-walk-test.
timeFrame: nine months
measure: 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.
timeFrame: nine months
measure: 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.
timeFrame: nine months
measure: 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.
timeFrame: nine months
measure: 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.
timeFrame: nine months
measure: 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.
timeFrame: nine months
Other outcomes:
Locations:
city: Munich
country: Germany
facility: Friedrich-Baur-Institute, Dep. of Neurology Klinikum der Universitaet Muenchen Munich, Germany
state: Bavaria
Study officials:
affiliation: Neurologist
name: Stephan Wenninger, Dr.med.
role: PRINCIPAL_INVESTIGATOR
References: