Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy

July 9, 2018 updated by: National Institute of Respiratory Diseases, Mexico

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

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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

Enrollment (Anticipated)

27

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Mexico
      • Mexico, Mexico, 14080
        • Recruiting
        • National Institute of Respiratory Diseases
        • Contact:
        • Principal Investigator:
          • Martha G Torres Fraga, MD
        • Sub-Investigator:
          • Jose L Carrillo Alduenda, MD
        • Sub-Investigator:
          • Luis Torre Bouscoulet, PhD
        • Sub-Investigator:
          • Oscar Hernandez Hernandez, PhD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Three months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Three months
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.
Three months
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.
three months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Institute of Respiratory Diseases, Mexico

Investigators

  • 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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 1, 2016

Primary Completion (Anticipated)

December 1, 2018

Study Completion (Anticipated)

April 1, 2019

Study Registration Dates

First Submitted

August 12, 2016

First Submitted That Met QC Criteria

August 22, 2016

First Posted (Estimate)

August 26, 2016

Study Record Updates

Last Update Posted (Actual)

July 11, 2018

Last Update Submitted That Met QC Criteria

July 9, 2018

Last Verified

July 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • C46-15

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Yes

IPD Plan Description

Participant data to be shared will be: Results of functional testing and prescription ventilation.

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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