Myotonic Dystrophy 1DM-IMTNCT04052958

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

A study testing IMT - inspiratory muscle training in people with Myotonic Dystrophy 1, run by LMU Klinikum at 1 site in Germany.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Myotonic Dystrophy 1
What is being tested?: IMT - inspiratory muscle training
Type of treatment: Medical device
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 45 participants
Where: 1 siteGermany
Study dates: 15 Aug 2019to 31 Dec 2021
Phase: Interventional
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

1 site in 1 country.

Germany1 site

Friedrich-Baur-Institute, Dep. of Neurology Klinikum der Universitaet Muenchen Munich, Germany
Munich, Bavaria, 80336

Other trials in Germany

About this study

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
Being tested: IMT - inspiratory muscle training
Medical subject headings: Myotonic Dystrophy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

IMT - inspiratory muscle trainingDevice
respiratory strength or indurance training with respiratory therapy device

Study groups in NCT04052958
Study group	Role	What they receive
Group 1: Strength Training	Active comparator	Respiratory muscle strength training Receives: Device: IMT - inspiratory muscle training
Group 2: Endurance Training	Active comparator	Respiratory muscle endurance training Receives: Device: IMT - inspiratory muscle training
Group 3: Control group	No intervention	no training of respiratory muscles

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: nine months

Secondary outcomes (20)

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).
Measured: nine months
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).
Measured: nine months
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).
Measured: nine months
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).
Measured: nine months
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).
Measured: nine months
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).
Measured: nine months
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).
Measured: nine months
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).
Measured: nine months
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.
Measured: nine months
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.
Measured: nine months
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.
Measured: nine months
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.
Measured: nine months
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.
Measured: nine months
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.
Measured: nine months
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.
Measured: nine months
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.
Measured: nine months
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.
Measured: nine months
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.
Measured: nine months
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.
Measured: nine months
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.
Measured: nine months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

15 Aug 201931 Dec 2021

First posted12 Aug 2019
Study start15 Aug 2019
Primary completion31 Dec 2021
Study completion31 Dec 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Myotonic Dystrophy 1Studies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase N/A
Primary purpose: Supportive care
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 45 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04052958
Sponsor's protocol ID: Version 1.2 (26. Juni 2019)
Acronym: DM-IMT
Status last verified: Apr 2022
First submitted: 31 Jul 2019
Last updated: 2 May 2022
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Myotonic Dystrophy 1 trials

Study at a glance

From the registry record, last updated by the sponsor on 2 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT04052958
Acronym / sponsor ID: DM-IMT / Version 1.2 (26. Juni 2019)
Conditions: Myotonic Dystrophy 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 45 participants (actual)
Lead sponsor: LMU KlinikumUniversity, hospital or other
Study start: 15 Aug 2019 (actual)
Primary completion: 31 Dec 2021 (actual)
Study completion: 31 Dec 2021 (actual)
First posted: 12 Aug 2019 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Supportive care
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 3

Arm	Type	Receives
Group 1: Strength Training	Active comparator	Device: IMT - inspiratory muscle trainingRespiratory muscle strength training
Group 2: Endurance Training	Active comparator	Device: IMT - inspiratory muscle trainingRespiratory muscle endurance training
Group 3: Control group	No intervention	–no training of respiratory muscles

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

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

Key exclusion criteria (8 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
IMT - inspiratory muscle trainingrespiratory strength or indurance training with respiratory therapy device	Device	Group 1: Strength Training, Group 2: Endurance Training

Primary outcome

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.

20 secondary outcomes

Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by MIP.
Time frame: nine months
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by MIP.
Time frame: nine months
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by MEP.
Time frame: nine months
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by MEP.
Time frame: nine months
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by FVC.
Time frame: nine months
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by FVC.
Time frame: nine months
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by FEV1.
Time frame: nine months
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by FEV1.
Time frame: nine months
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by pCO2.
Time frame: nine months
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by pCO2.
Time frame: nine months
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by pO2.
Time frame: nine months
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by pO2.
Time frame: nine months
Efficacy of recurrent inspiratory muscle strength training in 15 patients with type 1 myotonic dystrophy measured by pH.
Time frame: nine months
Efficacy of recurrent inspiratory muscle endurance training in 15 patients with type 1 myotonic dystrophy measured by pH.
Time frame: nine months
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
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 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 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 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 compared to patients without training, using the 6-minute-walk-test.
Time frame: nine months

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 2 May 2022.
Individual participant dataSharing planned: No

Related trials for Myotonic Dystrophy 1

RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
RecruitingObservational study
Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension
DM1, Myotonic Dystrophy
Enrolling by invitationPhase 2
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
Myotonic Dystrophy Type 1 (DM1)
RecruitingPhase 3
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
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RecruitingPhase 3
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1), DM1

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Stephan Wenninger, Dr.med. principal investigator

Neurologist

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A study testing IMT - inspiratory muscle training in people with Myotonic Dystrophy 1, run by LMU Klinikum at 1 site in Germany.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 2 May 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety of recurrent inspiratory muscle training (time frame: nine months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04052958.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 May 2022.