Amyotrophic Lateral SclerosisNCT00537641
Efficacy of Noninvasive Ventilation in Amyotrophic Lateral Sclerosis (ALS)
An observational study of Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by Columbia University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- May 2007to Jun 2011
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with ALS who are using nocturnal noninvasive ventilation to treat respiratory insufficiency will be screened for eligibility as below.
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Eleanor and Lou Gehrig ALS/MDA Center at Columbia University
New York, New York, 10032
About this study
Official title: Prevalence and Severity of Nocturnal Oxygenation and Ventilation Failure in Patients With Amyotrophic Lateral Sclerosis Using Noninvasive Ventilation
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.
Read the 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.
- Keywords
- noninvasive ventilation
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
What is being measured
The primary outcome is the main question the study is designed to answer.
Tolerance of bilevel PAP
The ALS Functional Rating Scale - Revised (ALSFRS-R) will be administered to measure tolerance.
Measured: Up to 3 months
Score on Epworth Sleepiness Scale
Objective evidence of nocturnal sleep-disordered breathing on the bilevel PAP will be measured.
Measured: Up to 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2007 (estimated)
- Study startMay 2007
- Primary completionJul 2010
- Study completionJun 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by Columbia UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Columbia University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Robert Basner, MD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00537641
- Sponsor's protocol ID
- AAAC2000
- Status last verified
- Jul 2015
- First submitted
- 27 Sep 2007
- Last updated
- 7 Jul 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prevalence and Severity of Nocturnal Oxygenation and Ventilation Failure in Patients With Amyotrophic Lateral Sclerosis Using Noninvasive Ventilation
- ClinicalTrials.gov ID
- NCT00537641
- Acronym / sponsor ID
- AAAC2000
- Keywords
- noninvasive ventilation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Columbia UniversityUniversity, hospital or other
- Study start
- May 2007
- Primary completion
- Jul 2010 (actual)
- Study completion
- Jun 2011 (actual)
- First posted
- 1 Oct 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with ALS who are using nocturnal noninvasive ventilation to treat respiratory insufficiency will be screened for eligibility as below.
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Tolerance of bilevel PAP
Time frame: Up to 3 months
The ALS Functional Rating Scale - Revised (ALSFRS-R) will be administered to measure tolerance.
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert Basner, MD principal investigator
Columbia University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by Columbia University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Jul 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Tolerance of bilevel PAP (time frame: Up to 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jul 2015.