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Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy

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ClinicalTrials.gov Identifier: NCT02880735
Recruitment Status : Recruiting
First Posted : August 26, 2016
Last Update Posted : July 11, 2018
Sponsor:
Information provided by (Responsible Party):
National Institute of Respiratory Diseases, Mexico

Tracking Information
First Submitted Date  ICMJE August 12, 2016
First Posted Date  ICMJE August 26, 2016
Last Update Posted Date July 11, 2018
Actual Study Start Date  ICMJE September 2016
Estimated Primary Completion Date December 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 22, 2016)
Ventilatory Response to Chemical Stimuli [ Time Frame: Three months ]
Increase the minute volume per unit of hypercapnia or hypoxemia, in a test of acute stimulation.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02880735 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 12, 2016)
  • Health Related Quality of Life Measured by Short Form 36 (SF-36) [ Time Frame: Three months ]
    Scale for measuring the quality of life related to health in 8 domains (vitality, physical functioning, bodily pain, general health perception, physical role functioning, emotional role functioning, social role functioning, mental health), each rated from 0 to 100%. The higher score means better quality of life.
  • Dyspnoea evaluated by the modified scale of the Medical Research Council (mMRC) [ Time Frame: Three months ]
    Rate dyspnea at 5 degrees from 0 to 5. 0: Not troubled by breathless except on strenuous exercise. 1: Short of breath when hurrying on a level or when walking up a slight hill. 2: Walks slower than most people on the level, stops after a mile or so, or stops after 15 minutes walking at own pace. 3: Stops for breath after walking 100 yards, or after a few minutes on level ground. 4: Too breathless to leave the house, or breathless when dressing/undressing.
  • Sleep Quality assessed by The Pittsburgh Sleep Quality Index (PSQI) [ Time Frame: three months ]
    Assesses sleep quality. Contains 19 questions, each weighted on a 0-3 interval scale. A global PSQI score is taken from the survey, with lower scores correlating to better sleep quality.
Original Secondary Outcome Measures  ICMJE
 (submitted: August 22, 2016)
  • Health Related Quality of Life Measured by Short Form 36 (SF-36) [ Time Frame: Three months ]
    Scale for measuring the quality of life related to health in 8 domains (vitality, physical functioning, bodily pain, general health perception, physical role functioning, emotional role functioning, social role functioning, mental health), each rated from 0 to 100%. The higher score means better quality of life.
  • Dyspnoea evaluated by the modified scale of the Medical Reserch Council (mMRC) [ Time Frame: Three months ]
    Rate dyspnea at 5 degrees from 0 to 5. 0: Not troubled by breathless except on strenuous exercise. 1: Short of breath when hurrying on a level or when walking up a slight hill. 2: Walks slower than most people on the level, stops after a mile or so, or stops after 15 minutes walking at own pace. 3: Stops for breath after walking 100 yards, or after a few minutes on level ground. 4: Too breathless to leave the house, or breathless when dressing/undressing.
  • Sleep Quality assessed by The Pittsburgh Sleep Quality Index (PSQI) [ Time Frame: three months ]
    Assesses sleep quality. Contains 19 questions, each weighted on a 0-3 interval scale. A global PSQI score is taken from the survey, with lower scores correlating to better sleep quality.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Myotonic Dystrophy 1
  • Steinert Disease
Intervention  ICMJE Device: Non Invasive Ventilation.
Non Invasive Mechanical Ventilation through oronasal mask. Mode: Bilevel with rate backup (spontaneous/time). Inspiratory Pressure: 30-10, Expiratory Pressure : 4-15, Backup Rate;14-25 rpm. Use Time: During Sleep.
Study Arms  ICMJE Experimental: Non Invasive Ventilation.

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.

Intervention: Device: Non Invasive Ventilation.
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 22, 2016)
27
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 2019
Estimated Primary Completion Date December 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Martha G Torres Fraga, MD (52) 5556668640 dra_marthagtf@hotmail.com
Contact: Jose L Carrillo Alduenda, MD (52)5554871700 ext 5242 jlcarrillo14@hotmail.com
Listed Location Countries  ICMJE Mexico
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02880735
Other Study ID Numbers  ICMJE C46-15
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: Participant data to be shared will be: Results of functional testing and prescription ventilation.
Responsible Party National Institute of Respiratory Diseases, Mexico
Study Sponsor  ICMJE National Institute of Respiratory Diseases, Mexico
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Chair: Rogelio Perez Padilla, MD National Institute of Respiratory Diseases, Mexico
Principal Investigator: Martha G Torres Fraga, MD National Institute of Respiratory Diseases, Mexico
PRS Account National Institute of Respiratory Diseases, Mexico
Verification Date July 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP