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

NCT04052958 | Completed

• 1 other identifier: Version 1.2 (26. Juni 2019)
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 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.

Conditions

Myotonic Dystrophy 1

Outcome Measures

Primary Outcomes (1)

• 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.

  nine months

Secondary Outcomes (20)

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

  nine months

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

  nine months

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

  nine months

• Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by MEP.

  nine months

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

  nine months

Study Arms (3)

Group 1: Strength Training

ACTIVE COMPARATOR

Respiratory muscle strength training

Device: IMT - inspiratory muscle training

Group 2: Endurance Training

ACTIVE COMPARATOR

Respiratory muscle endurance training

Device: IMT - inspiratory muscle training

Group 3: Control group

NO INTERVENTION

no training of respiratory muscles

Interventions

IMT - inspiratory muscle training DEVICE

respiratory strength or indurance training with respiratory therapy device

Group 1: Strength Training; Group 2: Endurance Training

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• LMU Klinikum: lead

Study Sites (1)

Friedrich-Baur-Institute, Dep. of Neurology Klinikum der Universitaet Muenchen Munich, Germany

Munich, Bavaria, 80336, Germany

Location

MeSH Terms

Conditions

Myotonic Dystrophy

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Myotonic Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Stephan Wenninger, Dr.med.

  Neurologist

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Neurologist, Senior Physician

Study Record Dates

• First Submitted: July 31, 2019
• First Posted: August 12, 2019
• Study Start: August 15, 2019
• Primary Completion: December 31, 2021
• Study Completion: December 31, 2021
• Last Updated: May 2, 2022

Record last verified: 2022-04

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)