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

Learn more about:
Related Clinical Trial
DMCRN-02-001: Assessing Pediatric Endpoints in DM1 Myotonic Dystrophy Type 1 and Resistance Exercise Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients Biomarker Development for Muscular Dystrophies Extracellular RNA Biomarkers of Myotonic Dystrophy Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1 Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1 Poor Neck Proprioception May Cause Balance Deficits in Myotonic Dystrophy 1 Myotonic Dystrophy – Vascular and Cognition Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1 Open Label Study in Adolescents and Children With Myotonic Disorders European Home Mechanical Ventilation Registry Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1) Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1 A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1 Sleep Breathing Disorders, a Main Trigger for Cardiac ARythmias in Type I Myotonic Dystrophy ? Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1 Effects of a 12-week Strength Training Program in Men With Myotonic Dystrophy Type 1 Efficacy and Safety of DHEA for Myotonic Dystrophy Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy Effects of a Multiple Component Training Program on Muscles in Adults With Myotonic Dystrophy Type 1 Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1 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 Venous Thromboembolism in DM1 Postural Spirometry Changes in Ambulatory Myotonic Dystrophy Patients Factors Associated With Hypoventilation in the Myotonic Dystrophy, Progressive Profile Over 5 Years PhenoDM1 (Myotonic Dystrophy Type 1 Natural History Study) Children’s Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Observational Prolonged Trial in Myotonic Dystrophy Type 1 Venous Thromboembolism in Myotonic Dystrophy Type 1 Myotonic Dystrophy Type 1 Aerobic Exercise Study Multicenter Observational Study of Myotonic Dystrophy Type 1 DM1 Heart Registry – DM1 Respiratory Registry

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

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

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.
    



Study Type

Interventional


Primary Outcome

Safety of recurrent inspiratory muscle training

Secondary Outcome

 Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by MIP.

Condition

Myotonic Dystrophy 1

Intervention

IMT - inspiratory muscle training

Study Arms / Comparison Groups

 Group 1: Strength Training
Description:  Respiratory muscle strength training

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Device

Estimated Enrollment

45

Start Date

August 15, 2019

Completion Date

December 31, 2021

Primary Completion Date

December 31, 2021

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
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Stephan Wenninger, Dr.med., , 

Location Countries

Germany

Location Countries

Germany

Administrative Informations


NCT ID

NCT04052958

Organization ID

Version 1.2 (26. Juni 2019)


Responsible Party

Principal Investigator

Study Sponsor

LMU Klinikum


Study Sponsor

Stephan Wenninger, Dr.med., Principal Investigator, Neurologist


Verification Date

April 2022