Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy

NCT02880735 | Unknown

• 1 other identifier: C46-15
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 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.

Conditions

Myotonic Dystrophy 1; Steinert Disease

Keywords

Myotonic Dystrophy; Noninvasive Ventilation; Respiratory Failure

Outcome Measures

Primary Outcomes (1)

• Ventilatory Response to Chemical Stimuli

  Increase the minute volume per unit of hypercapnia or hypoxemia, in a test of acute stimulation.

  Three months

Secondary Outcomes (3)

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

  Three months

• Dyspnoea evaluated by the modified scale of the Medical Research Council (mMRC)

  Three months

• Sleep Quality assessed by The Pittsburgh Sleep Quality Index (PSQI)

  three months

Study Arms (1)

Non Invasive Ventilation.

EXPERIMENTAL

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.

Interventions

Non Invasive Ventilation. DEVICE

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.

Non Invasive Ventilation.

Eligibility Criteria

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

You may qualify if:

• Classic variety of Myotonic Dystrophy Type 1
• Molecular confirmation through the standard method

You may not qualify if:

• 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

Sponsors & Collaborators

• National Institute of Respiratory Diseases, Mexico: lead

Study Sites (1)

National Institute Of Respiratory Diseases

México, 14080, Mexico

RECRUITING

Related Publications (7)

• Meola G. Clinical aspects, molecular pathomechanisms and management of myotonic dystrophies. Acta Myol. 2013 Dec;32(3):154-65.

  PMID: 24803843 RESULT

• Mankodi A, Thornton CA. Myotonic syndromes. Curr Opin Neurol. 2002 Oct;15(5):545-52. doi: 10.1097/00019052-200210000-00005.

  PMID: 12351998 RESULT

• Carroll JE, Zwillich CW, Weil JV. Ventilatory response in myotonic dystrophy. Neurology. 1977 Dec;27(12):1125-8. doi: 10.1212/wnl.27.12.1125.

  PMID: 563010 RESULT

• Poussel M, Thil C, Kaminsky P, Mercy M, Gomez E, Chaouat A, Chabot F, Chenuel B. Lack of correlation between the ventilatory response to CO2 and lung function impairment in myotonic dystrophy patients: evidence for a dysregulation at central level. Neuromuscul Disord. 2015 May;25(5):403-8. doi: 10.1016/j.nmd.2015.02.006. Epub 2015 Feb 17.

  PMID: 25753091 RESULT

• Jammes Y, Pouget J, Grimaud C, Serratrice G. Pulmonary function and electromyographic study of respiratory muscles in myotonic dystrophy. Muscle Nerve. 1985 Sep;8(7):586-94. doi: 10.1002/mus.880080708.

  PMID: 4047090 RESULT

• Brooks D, De Rosie J, Mousseau M, Avendano M, Goldstein RS. Long term follow-up of ventilated patients with thoracic restrictive or neuromuscular disease. Can Respir J. 2002 Mar-Apr;9(2):99-106. doi: 10.1155/2002/545612.

  PMID: 11972163 RESULT

• Annane D, Quera-Salva MA, Lofaso F, Vercken JB, Lesieur O, Fromageot C, Clair B, Gajdos P, Raphael JC. Mechanisms underlying effects of nocturnal ventilation on daytime blood gases in neuromuscular diseases. Eur Respir J. 1999 Jan;13(1):157-62. doi: 10.1183/09031936.99.13115799.

  PMID: 10836341 RESULT

MeSH Terms

Conditions

Myotonic Dystrophy; Respiratory Insufficiency

Interventions

Noninvasive Ventilation

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; Respiration Disorders; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Respiration, Artificial; Airway Management; Therapeutics; Respiratory Therapy

Study Officials

• Rogelio Perez Padilla, MD

  National Institute of Respiratory Diseases, Mexico

  STUDY CHAIR

• Martha G Torres Fraga, MD

  National Institute of Respiratory Diseases, Mexico

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Martha G Torres Fraga, MD

CONTACT

(52) 5556668640; dra_marthagtf@hotmail.com

Jose L Carrillo Alduenda, MD

CONTACT

(52)5554871700; jlcarrillo14@hotmail.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 12, 2016
• First Posted: August 26, 2016
• Study Start: September 1, 2016
• Primary Completion: December 1, 2018
• Study Completion: April 1, 2019
• Last Updated: July 11, 2018

Record last verified: 2018-07

Data Sharing

• IPD Sharing: Will share

Locations

ME (1)