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| Sponsored by: |
University Hospital, Grenoble |
|---|---|
| Information provided by: | University Hospital, Grenoble |
| ClinicalTrials.gov Identifier: | NCT00745238 |
Purpose
Background: Myotonic dystrophy lead to highly heterogeneous, multisystemic symptoms including myotonia, progressive muscle weakness, cardiac conduction defects, cataract, metabolic dysfunction, and excessive daytime somnolence. This last symptom is related to respiratory failure and/or to involvement of the central nervous system. However the metabolic disturbances could contribute to it. From the respiratory point of view this disease is characterised by the progressive appearance of respiratory failure of muscular origin but mainly associated with a defect in the central respiratory drive. The treatment for this hypoventilation is non-invasive ventilation (NIV).
It is not currently absolutely clear as to the best choice of criteria to judge long term effectiveness of NIV. The most usual criteria are normalisation of daytime blood gases, diminution of respiratory work, improvement in daytime symptoms and improvement in sleep structure. Other criteria are currently little studied, for instance the contribution of the interaction between alveolar hypoventilation and oxygen desaturation during the night and biological deficiencies such as systemic inflammation, glucose intolerance or insulin resistance. Likewise there is little information about the interaction between alveolar hypoventilation and endothelial dysfunction and arterial stiffness both being accurate predictive factors for cardiovascular risks.
Aim: to evaluate the impact of NIV on endothelial dysfunction in patients with myotonic dystrophy. The secondary objectives are to assess the impact of NIV on systemic inflammation, arterial stiffness, insulin-resistance, quality of sleep, and daytime vigilance in these patients.
Methods: Patients with chronic alveolar hypoventilation already treated by long term NIV will be included. They will have an initial check-up (Visit 1), then will interrupt NIV treatment for four weeks (Visit 2), and then return to NIV treatment. The last check-up will be done four weeks after NIV resumption (Visit3).
Expected results: It is expected that NIV withdrawal will results in a deterioration of cardio-vascular parameters (endothelial function and arterial stiffness), metabolic parameters (insulin-resistance and systemic inflammation), quality of sleep and daytime vigilance. Return to NIV treatment may show an improvement of these parameters with a basal state recovery.
| Condition | Intervention |
|---|---|
|
Myotonia |
Other: withdrawal of non-invasive ventilation |
| Study Type: | Interventional |
| Study Design: | Supportive Care, Open Label, Active Control, Single Group Assignment, Efficacy Study |
| Official Title: | Four Weeks Withdrawal of Non-Invasive Ventilation (NIV) in Patients With Myotonic Dystrophy: Cardiovascular, Metabolic and Daytime Vigilance Induced Changes |
| Estimated Enrollment: | 35 |
| Study Start Date: | June 2008 |
| Estimated Study Completion Date: | June 2010 |
| Estimated Primary Completion Date: | June 2010 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Myotonic Dystrophy 1: Experimental |
Other: withdrawal of non-invasive ventilation
Four weeks withdrawal of non-invasive ventilation
|
NIV is a technique of assisted ventilation that does not use the endotracheal route as the interface between the patient and the ventilator. NIV by positive pressure assistance involves ventilating the patient by means of a mask adjusted on the nose or covering the nose and mouth. This technique is now the recommended therapeutic strategy for the treatment of chronic alveolar hypoventilation. It improves survival and quality of life, and improves daytime blood gases in patients suffering form chronic restrictive respiratory failure.
For each check-up (3 visits), patients will have a polysomnography, a complete respiratory function measurement (Flows and lung volumes, CO2 sensitivity test, SNIF test, blood gazes analysis), a test of endothelial function (peripheral arterial tone), a test of arterial stiffness (pulse wave velocity), an assessment of systemic inflammation (ultra sensitive CRP, TNFa, IL6), assessment of diurnal vigilance tests ( OSLER test and sleepiness scale) and assessment of metabolic and endocrinal function (insulinemia, glucose blood level, Leptin, DHEA).
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
INCLUSION CRITERIA:
EXCLUSION CRITERIA:
Contacts and Locations| Contact: Jean Louis PEPIN JP PEPIN, PROFESSOR | 00330476765516 | JPepin@chu-grenoble.fr |
| France, ISERE | |
| France.Functional Cardio-Respiratory Exploration Laboratory | Recruiting |
| GRENOBLE., ISERE, France, 38000 | |
| Contact: Jean -Louis JL PEPIN, professor 0033476765516 JPepin@chu-grenoble.fr | |
| Principal Investigator: | Jean Louis PEPIN, PROFESSOR | CHU grenoble |
More Information
| Responsible Party: | University Hospital of Grenoble ( University Hospital of Grenoble ) |
| Study ID Numbers: | 0801 |
| Study First Received: | September 1, 2008 |
| Last Updated: | September 2, 2008 |
| ClinicalTrials.gov Identifier: | NCT00745238 |
| Health Authority: | France: Afssaps - French Health Products Safety Agency |
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endothelial function peripheral arterial tone systemic inflammation oxidative stress respiratory and peripheral muscular function |
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Stress Dystrophia myotonica 1 Neurodegenerative Diseases Myotonia Myotonic Disorders Inflammation Myotonia atrophica Signs and Symptoms Muscular Dystrophies Muscular Diseases |
Heredodegenerative Disorders, Nervous System Genetic Diseases, Inborn Neuromuscular Diseases Muscular Disorders, Atrophic Musculoskeletal Diseases Myotonic Dystrophy Neurologic Manifestations Atrophy Muscular dystrophy |
|
Neuromuscular Manifestations Nervous System Diseases |
