Amyotrophic Lateral Sclerosis (ALS)NCT01363882
Polysomnography-directed Noninvasive Ventilation in Amyotrophic Lateral Sclerosis (ALS)
A study testing Sleep study-guided adjustment of NIV and Standard initiation of NIV in people with Amyotrophic Lateral Sclerosis (ALS), run by Columbia University at 1 site in United States.
Trial summary
Use of noninvasive ventilation (NIV, also known colloquially as "Bipap") has been associated in some studies with improvement in pulmonary function, quality of life and survival. NIV is typically applied during sleep, and without the benefit of sleep study to determine the optimal settings. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- Sleep study-guided adjustment of NIV and Standard initiation of NIV
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2008to Jul 2010
- Phase
- Interventional
- 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
Who may be able to join
- Diagnosis of probable or definite Amyotrophic Lateral Sclerosis (ALS) per El Escorial criteria
- Between ages of 18 and 80 yrs old
Who cannot join
- Inability to clear secretions from the airway
- Life expectancy < 6 months from a comorbid condition
- Dementia sufficient to impair ability to use NIV, perform respiratory muscle pressure testing (PFTs), or complete Health-related Quality of Life (HRQOL) instruments
- Inability to follow up at the ALS Center on a regular basis
- Previously diagnosed obstructive Sleep Apnea
Trial locations and contacts
1 site in 1 country.
United States1 site
Columbia University Medical Center
New York, New York, 10032
About this study
Official title: Progression of Respiratory Dysfunction in Amyotrophic Lateral Sclerosis (ALS) Patients: A Comparison of Standard of Practice vs Polysomnography-Directed Nocturnal Non-Invasive Positive Pressure Ventilation
Use of noninvasive ventilation (NIV, also known colloquially as "Bipap") has been associated in some studies with improvement in pulmonary function, quality of life and survival. NIV is typically applied during sleep, and without the benefit of sleep study to determine the optimal settings. The investigators have shown that when NIV is used in this fashion, failure of nocturnal oxygenation and ventilation is prominent. This study is randomizing patients to standard application of NIV vs application guided by use of sleep study data to determine the effect of titrated therapy on pulmonary function, quality of life and survival.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- Sleep study-guided adjustment of NIV, Standard initiation of NIV
- Keywords
- Amyotrophic Lateral Sclerosis, Noninvasive Ventilation, Respiratory Dysfunction, Positive Pressure Ventilation
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Dyspnea
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sleep study-guided adjustment of NIVOther
Sleep studies will be performed at baseline, within 2 weeks to initially titrate NIV, and at 1, 3 and 6 months to assess NIV performance and adjust it as necessary based on oxygenation and ventilation parameters.
Standard initiation of NIVOther
NIV will be initiated and managed as per current standard of practice. Sleep studies will be performed at baseline, 2 weeks, 1, 3 and 6 months to gather data but will not influence NIV management. NIV will be adjusted by a respiratory therapist or the subject's primary physician per waking symptoms.
| Study group | Role | What they receive |
|---|---|---|
| Standard NIV | Active comparator | Noninvasive ventilation (NIV) will be initiated and managed as per current standard of practice guided by the American Academy of Neurology (AAN) Practice Parameters (updated in 2009), in all subjects with amyotrophic lateral sclerosis (ALS) and a forced vital capacity of <50% predicted. Sleep studies will be performed at baseline, 2 weeks, 1, 3 and 6 months, but will not influence management of the NIV. Receives: Other: Standard initiation of NIV |
| Sleep study titrated NIV | Experimental | ALS subjects in this arm, who are offered NIV for Forced Vital Capacity (FVC) <50% as per AAN Practice Parameters, will have their initial level of NIV determined polysomnographically. They will be followed with sleep studies at 1 month, 3 months and 6 months to reassess NIV efficacy and NIV will be adjusted as necessary to optimize parameters of oxygenation and ventilation. Receives: Other: Sleep study-guided adjustment of NIV |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in spirometric, respiratory muscle strength, and gas exchange measures
FVC (forced vital capacity), FEV1 (forced expiratory volume in 1 second), MIP (maximum inspiratory pressure) and MEP (maximum expiratory pressure
Measured: Up to 6 months after starting NIV
Secondary outcomes (4)
Duration that the Mental Component Summary (MCS) is maintained above 75% of baseline score for the Medical Outcomes Study Health Survey (SF-12)
Measured: Up to 6 months after starting NIV
Survival
Whether sleep study-titrated NIV is associated with trends to improved survival, compared with standardly prescribed (non-titrated) NIV
Measured: Up to 6 months after baseline
Nocturnal oxygenation and ventilation
Nadir oxygen saturation, number of oxygen desaturations of 3%/hr (ODI3), % time of sleep spent with oxygen saturation <90%, apnea-hypopnea index, asynchrony index
Measured: Up to 6 months after starting NIV
Modified Borg dyspnea score (see description)
The Modified Borg Dyspnea Scale is a 10-point Likert scale asking subjects to rate perceived shortness of breath, ranging from 0 (no breathlessness at all) to 10 (maximal breathlessness)
Measured: Up to 6 months after baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jun 2011 (estimated)
- Study startFeb 2008
- Primary completionJul 2010
- Study completionJul 2010
Why it stopped: Insufficient enrollment \& high rate of failure to complete protocol
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Columbia University
- Sponsor type
- University, hospital or other
- Collaborators
- ALS Association
- Responsible party
- Sponsor
- Investigators
- Robert C Basner, MD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01363882
- Sponsor's protocol ID
- AAAC6753
- Status last verified
- Nov 2020
- First submitted
- 28 Mar 2011
- Last updated
- 13 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Progression of Respiratory Dysfunction in Amyotrophic Lateral Sclerosis (ALS) Patients: A Comparison of Standard of Practice vs Polysomnography-Directed Nocturnal Non-Invasive Positive Pressure Ventilation
- ClinicalTrials.gov ID
- NCT01363882
- Acronym / sponsor ID
- AAAC6753
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Keywords
- Amyotrophic Lateral Sclerosis, Noninvasive Ventilation, Respiratory Dysfunction, Positive Pressure Ventilation
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Insufficient enrollment \& high rate of failure to complete protocol
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Columbia UniversityUniversity, hospital or other
- Collaborators
- ALS Association
- Study start
- Feb 2008
- Primary completion
- Jul 2010 (actual)
- Study completion
- Jul 2010 (actual)
- First posted
- 2 Jun 2011 (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.
Use of noninvasive ventilation (NIV, also known colloquially as "Bipap") has been associated in some studies with improvement in pulmonary function, quality of life and survival. NIV is typically applied during sleep, and without the benefit of sleep study to determine the optimal settings. The investigators have shown that when NIV is used in this fashion, failure of nocturnal oxygenation and ventilation is prominent. This study is randomizing patients to standard application of NIV vs application guided by use of sleep study data to determine the effect of titrated therapy on pulmonary function, quality of life and survival.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Standard NIV | Active comparator | Other: Standard initiation of NIVNoninvasive ventilation (NIV) will be initiated and managed as per current standard of practice guided by the American Academy of Neurology (AAN) Practice Parameters (updated in 2009), in all subjects with amyotrophic lateral sclerosis (ALS) and a forced vital capacity of <50% predicted. Sleep studies will be performed at baseline, 2 weeks, 1, 3 and 6 months, but will not influence management of the NIV. |
| Sleep study titrated NIV | Experimental | Other: Sleep study-guided adjustment of NIVALS subjects in this arm, who are offered NIV for Forced Vital Capacity (FVC) <50% as per AAN Practice Parameters, will have their initial level of NIV determined polysomnographically. They will be followed with sleep studies at 1 month, 3 months and 6 months to reassess NIV efficacy and NIV will be adjusted as necessary to optimize parameters of oxygenation and ventilation. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Diagnosis of probable or definite Amyotrophic Lateral Sclerosis (ALS) per El Escorial criteria
- Between ages of 18 and 80 yrs old
Key exclusion criteria (5 listed)
- Inability to clear secretions from the airway
- Life expectancy < 6 months from a comorbid condition
- Dementia sufficient to impair ability to use NIV, perform respiratory muscle pressure testing (PFTs), or complete Health-related Quality of Life (HRQOL) instruments
- Inability to follow up at the ALS Center on a regular basis
- Previously diagnosed obstructive Sleep Apnea
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Sleep study-guided adjustment of NIVSleep studies will be performed at baseline, within 2 weeks to initially titrate NIV, and at 1, 3 and 6 months to assess NIV performance and adjust it as necessary based on oxygenation and ventilation parameters. | Other | Sleep study titrated NIV |
| Standard initiation of NIVNIV will be initiated and managed as per current standard of practice. Sleep studies will be performed at baseline, 2 weeks, 1, 3 and 6 months to gather data but will not influence NIV management. NIV will be adjusted by a respiratory therapist or the subject's primary physician per waking symptoms. | Other | Standard NIV |
Primary outcome
Change from baseline in spirometric, respiratory muscle strength, and gas exchange measures
Time frame: Up to 6 months after starting NIV
FVC (forced vital capacity), FEV1 (forced expiratory volume in 1 second), MIP (maximum inspiratory pressure) and MEP (maximum expiratory pressure
4 secondary outcomes
Duration that the Mental Component Summary (MCS) is maintained above 75% of baseline score for the Medical Outcomes Study Health Survey (SF-12)
Time frame: Up to 6 months after starting NIV
Survival
Time frame: Up to 6 months after baseline
Nocturnal oxygenation and ventilation
Time frame: Up to 6 months after starting NIV
Modified Borg dyspnea score (see description)
Time frame: Up to 6 months after baseline
Results and safety
The study was stopped early (reason given: Insufficient enrollment \& high rate of failure to complete protocol). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Nov 2020.
Related trials for Amyotrophic Lateral Sclerosis (ALS)
RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruiting
A Study on the Addition of Alsum30 for the Treatment of Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS), Bacillus Coagulans
Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
Neurologic Disorder, Neurologic Diseases
Not yet recruitingPhase 1
Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
Recruiting
Clinical Outcome Assessment for AT & BCI
Spinal Cord Injury, ALS (Amyotrophic Lateral Sclerosis)
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 C 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?
A study testing Sleep study-guided adjustment of NIV and Standard initiation of NIV in people with Amyotrophic Lateral Sclerosis (ALS), 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 2 inclusion and 5 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: stopped early, as of the sponsor's last update on 13 Nov 2020. 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 was stopped early (reason given: Insufficient enrollment \& high rate of failure to complete protocol). Results are not available in the source record.
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?
Change from baseline in spirometric, respiratory muscle strength, and gas exchange measures (time frame: Up to 6 months after starting NIV). 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 13 Nov 2020.