Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy

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

Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy

Official Title

Effect of Noninvasive Mechanical Ventilation on Ventilatory Response in Patients With Myotonic Dystrophy Type 1

Brief Summary

      It has been suggested that patients with Myotonic Dystrophy type 1 have primary altered
      ventilatory response to chemical stimuli and chronic hypoventilation is related not always to
      muscle weakness. Also, it is known that Non Invasive Mechanical Ventilation can improve
      ventilatory response to chemical stimuli, especially to hypercapnia.

      This study evaluates the effect of Non Invasive Mechanical Ventilation on ventilatory
      response in patients with Type 1 Myotonic Dystrophy, the ventilatory response to chemical
      stimuli will be measured before and after mechanical ventilation in patients with myotonic
      dystrophy type 1.
    

Detailed Description

      Type 1 Myotonic Dystrophy is a hereditary neuromuscular disease with an autosomal dominant
      pattern whose prevalence is 1/8000 inhabitants and is the most common muscular dystrophy in
      adults. It is multisystem disease and is characterized by myotonia, progressive muscle loss
      and a wide spectrum of manifestations.

      Myotonic dystrophy type 1 causes a high impact on health and quality of life of patients as
      functional impairment can reach the incapacity and total dependence in basic activities of
      daily living. As in most neuromuscular diseases, progressive muscle weakness at some point in
      the evolution affects the respiratory muscles. However, in some patients with myotonic
      dystrophy type 1 it has been observed that muscle weakness does not explain ventilatory
      failure, and is believed to be due to a primary reduction in the central ventilatory response
      to hypercapnia present in this disease.

      Non Invasive Mechanical Ventilation (NIV) is a long-term treatment that provides ventilatory
      assistance through an interface that does not invade the airway and currently can be provided
      to patients in the home environment; It is a resource that has shown to improve the quality
      of life, daytime gas exchange and survival in patients with neuromuscular diseases, even when
      used only during sleep. It is not clear the mechanism by which NIV during daytime sleep
      improves gas exchange in patients with neuromuscular diseases, even in advanced stages where
      breathing muscles effectors are severely affected.

      It has been proposed that NIV used during sleep can improve the sensitivity of the
      respiratory center to carbon dioxide but this has not been demonstrated in patients with Type
      1 Myotonic Dystrophy, to answer this question, it is proposed to compare the central
      ventilatory response to chemical stimuli after a period of NIV in patients with Type 1
      Myotonic Dystrophy.
    


Study Type

Interventional


Primary Outcome

Ventilatory Response to Chemical Stimuli

Secondary Outcome

 Health Related Quality of Life Measured by Short Form 36 (SF-36)

Condition

Myotonic Dystrophy 1

Intervention

Non Invasive Ventilation.

Study Arms / Comparison Groups

 Non Invasive Ventilation.
Description:  It will provide noninvasive mechanical ventilation with the following specifications:
Bilevel devices: Pressurized bilevel mode Spontaneous/Time. Interface: Facial mask Usage: During sleep Frequency: Daily Duration: Three months.

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

27

Start Date

September 2016

Completion Date

April 2019

Primary Completion Date

December 2018

Eligibility Criteria

        Inclusion Criteria:

          -  Classic variety of Myotonic Dystrophy Type 1

          -  Molecular confirmation through the standard method

        Exclusion Criteria:

          -  Using currently invasive mechanical ventilation.

          -  Acute decompensation of respiratory or cardiac origin in the last 6 months, which
             required hospital care.

          -  Drugs that may alter the ventilatory response: benzodiazepines, neuroleptics,
             corticosteroids, theophylline, acetazolamide
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Rogelio Perez Padilla, MD, (52) 5556668640, [email protected]

Location Countries

Mexico

Location Countries

Mexico

Administrative Informations


NCT ID

NCT02880735

Organization ID

C46-15


Responsible Party

Sponsor

Study Sponsor

National Institute of Respiratory Diseases, Mexico


Study Sponsor

Rogelio Perez Padilla, MD, Study Chair, National Institute of Respiratory Diseases, Mexico


Verification Date

July 2018