Back to resultsPolysomnography-directed Noninvasive Ventilation in Amyotrophic Lateral Sclerosis (ALS)
Primary Purpose
Amyotrophic Lateral Sclerosis (ALS)
Status
Terminated
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Sleep study-guided adjustment of NIV
Standard initiation of NIV
Sponsored by
About this trial
This is an interventional supportive care trial for Amyotrophic Lateral Sclerosis (ALS) focused on measuring Amyotrophic Lateral Sclerosis, Noninvasive Ventilation, Respiratory Dysfunction, Positive Pressure Ventilation
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of probable or definite Amyotrophic Lateral Sclerosis (ALS) per El Escorial criteria
- Between ages of 18 and 80 yrs old
Exclusion Criteria:
- 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
Sites / Locations
- Columbia University Medical Center
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Experimental
Arm Label
Standard NIV
Sleep study titrated NIV
Arm Description
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.
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.
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
Duration that the Mental Component Summary (MCS) is maintained above 75% of baseline score for the Medical Outcomes Study Health Survey (SF-12)
Survival
Whether sleep study-titrated NIV is associated with trends to improved survival, compared with standardly prescribed (non-titrated) NIV
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
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)
Full Information
NCT ID
NCT01363882
First Posted
March 28, 2011
Last Updated
November 10, 2020
Sponsor
Columbia University
Collaborators
ALS Association
1. Study Identification
Unique Protocol Identification Number
NCT01363882
Brief Title
Polysomnography-directed Noninvasive Ventilation in Amyotrophic Lateral Sclerosis (ALS)
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
Study Type
Interventional
2. Study Status
Record Verification Date
November 2020
Overall Recruitment Status
Terminated
Why Stopped
Insufficient enrollment & high rate of failure to complete protocol
Study Start Date
February 2008 (undefined)
Primary Completion Date
July 2010 (Actual)
Study Completion Date
July 2010 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Columbia University
Collaborators
ALS Association
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief 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. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis (ALS)
Keywords
Amyotrophic Lateral Sclerosis, Noninvasive Ventilation, Respiratory Dysfunction, Positive Pressure Ventilation
7. Study Design
Primary Purpose
Supportive Care
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
25 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Standard NIV
Arm Type
Active Comparator
Arm Description
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.
Arm Title
Sleep study titrated NIV
Arm Type
Experimental
Arm Description
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.
Intervention Type
Other
Intervention Name(s)
Sleep study-guided adjustment of NIV
Intervention Description
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.
Intervention Type
Other
Intervention Name(s)
Standard initiation of NIV
Intervention Description
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.
Primary Outcome Measure Information:
Title
Change from baseline in spirometric, respiratory muscle strength, and gas exchange measures
Description
FVC (forced vital capacity), FEV1 (forced expiratory volume in 1 second), MIP (maximum inspiratory pressure) and MEP (maximum expiratory pressure
Time Frame
Up to 6 months after starting NIV
Secondary Outcome Measure Information:
Title
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
Title
Survival
Description
Whether sleep study-titrated NIV is associated with trends to improved survival, compared with standardly prescribed (non-titrated) NIV
Time Frame
Up to 6 months after baseline
Title
Nocturnal oxygenation and ventilation
Description
Nadir oxygen saturation, number of oxygen desaturations of 3%/hr (ODI3), % time of sleep spent with oxygen saturation <90%, apnea-hypopnea index, asynchrony index
Time Frame
Up to 6 months after starting NIV
Title
Modified Borg dyspnea score (see description)
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)
Time Frame
Up to 6 months after baseline
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of probable or definite Amyotrophic Lateral Sclerosis (ALS) per El Escorial criteria
Between ages of 18 and 80 yrs old
Exclusion Criteria:
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Robert C Basner, MD
Organizational Affiliation
Columbia University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Polysomnography-directed Noninvasive Ventilation in Amyotrophic Lateral Sclerosis (ALS)
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