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Efficacy of Noninvasive Ventilation in Amyotrophic Lateral Sclerosis (ALS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00537641
Recruitment Status : Completed
First Posted : October 1, 2007
Last Update Posted : July 7, 2015
Information provided by (Responsible Party):
Columbia University

Brief Summary:
This study will test the hypothesis that noninvasive ventilation (NIV) as prescribed in current medical practice for use in amyotrophic Lateral Sclerosis (ALS) patients fails to deliver adequate breathing support over a night of use in the patient's home. ALS patients who come to the ALS Center for their routine 3 month follow up exam and are currently using NIV will be asked to complete questionnaires regarding their quality of sleep, quality of life and general level of function, and to undergo a home sleep study, using a safe, comfortable and reliable breathing monitoring system during a night of sleep. If the questionnaires or the sleep study show failure of the breathing device, the investigators will work with the patient to fix the problem and then offer a second study to make sure that the changes were helpful. The results of this study may help to develop subsequent studies and to improve the guidelines used for care of ALS patients.

Condition or disease
Amyotrophic Lateral Sclerosis Motor Neuron Disease

Detailed Description:
Amyotrophic Lateral Sclerosis (ALS), also known as "Lou Gehrig's Disease", is a fatal disorder that causes breathing failure due to progressive weakness of the muscles of breathing. Breathing assist devices known as noninvasive ventilation (NIV) are offered to ALS patients when their breathing function worsens. These devices deliver breathing assistance via a mask on the nose or nose and mouth, and are thought to be particularly important to be used during sleep, when breathing often becomes more shallow and irregular. However, although these devices have become the standard of therapy in ALS patients once their lung function worsens, it remains unclear how effective these devices actually are when a patient is sleeping, partly because of the practical difficulties in applying the device properly and keeping it applied throughout the sleep period, and partly because they are most commonly prescribed without objective evidence regarding how much breathing support the patient needs as the disease progresses and the breathing muscles weaken further.

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Study Type : Observational
Actual Enrollment : 25 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Prevalence and Severity of Nocturnal Oxygenation and Ventilation Failure in Patients With Amyotrophic Lateral Sclerosis Using Noninvasive Ventilation
Study Start Date : May 2007
Actual Primary Completion Date : July 2010
Actual Study Completion Date : June 2011

Primary Outcome Measures :
  1. Tolerance of bilevel PAP [ Time Frame: Up to 3 months ]
    The ALS Functional Rating Scale — Revised (ALSFRS-R) will be administered to measure tolerance.

  2. Score on Epworth Sleepiness Scale [ Time Frame: Up to 3 months ]
    Objective evidence of nocturnal sleep-disordered breathing on the bilevel PAP will be measured.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with ALS who are using nocturnal noninvasive ventilation to treat respiratory insufficiency will be screened for eligibility as below.

Inclusion Criteria:

  • clinical diagnosis of amyotrophic lateral sclerosis (El Escorial criteria)
  • age 18 to 18 years old
  • using nocturnal noninvasive ventilation to treat respiratory insufficiency at least 4 hours per night, for at least 4 nights per week

Exclusion Criteria:

  • inability to safely use NIPPV because of bulbar dysfunction
  • indications for tracheostomy assisted ventilation due to inability to clear secretions from the airway
  • presence of comorbid conditions with a life expectancy < 6 months
  • presence of advanced dementia
  • unwillingness to follow up at the Eleanor and Lou Gehrig ALS/MDA Center at Columbia University on a regular basis
  • previously diagnosed obstructive sleep apnea
  • residence outside the New York metropolitan area.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00537641

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United States, New York
Eleanor and Lou Gehrig ALS/MDA Center at Columbia University
New York, New York, United States, 10032
Sponsors and Collaborators
Columbia University
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Principal Investigator: Robert Basner, MD Columbia University

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Responsible Party: Columbia University Identifier: NCT00537641    
Other Study ID Numbers: AAAC2000
First Posted: October 1, 2007    Key Record Dates
Last Update Posted: July 7, 2015
Last Verified: July 2015
Keywords provided by Columbia University:
noninvasive ventilation
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases