Effect of Noninvasive Ventilation on Lung Function in Amyotrophic Lateral Sclerosis

Effect of Noninvasive Positive Pressure Ventilation on Pulmonary Function Testing in Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis (ALS), or "Lou Gehrig's Disease", is a fatal disorder that causes progressive degeneration and weakening of the muscles of breathing, leading to breathing insufficiency and eventually breathing failure. This breathing insufficiency is commonly treated with a breathing assistance device, known as noninvasive positive pressure ventilation (NIPPV). While generally well tolerated and accepted, it is not clear whether or to what extent NIPPV in fact helps breathing function: some data suggest that NIPPV preserves breathing function over time, whereas other data suggest that it actually causes breathing function to decline more quickly. No studies have shown what the acute effect of NIPPV is on breathing muscle function in ALS patients.

This study will test the hypothesis that the acute use of NIPPV, at pressure levels that are in common clinical use, will cause measurable changes in tests of breathing function, compared to baseline and to lower levels of NIPPV. We expect that the results of this study will help to clarify whether and to what extent NIPPV assists respiratory muscle function in patients with ALS.

Study Overview

• Status: Terminated
• Conditions: Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Device: noninvasive positive pressure ventilation; Device: noninvasive positive pressure ventilation

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10032
      • Eleanor and Lou Gehrig ALS/MDA Center at Columbia University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• clinical diagnosis of amyotrophic lateral sclerosis
• clinical indication to start noninvasive ventilation (forced vital capacity < 50% predicted or signs/symptoms of respiratory insufficiency)
• age 18 to 80 years old

Exclusion Criteria:

• prior institution of NIPPV
• inability to safely use NIPPV
• indications for tracheostomy assisted ventilation because of inability to clear secretions from the airway
• inability or unwillingness to perform pulmonary function testing
• presence of advanced dementia.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: High-level ventilation; Each subject will spend 2 hours receiving high-level noninvasive ventilation.	Device: noninvasive positive pressure ventilation; Each subject will undergo 2 hours of high-level noninvasive positive pressure ventilation, with an inspiratory positive airway pressure of 12 cm H2O and an expiratory positive airway pressure of 3 cm H2O.; Other Names: "BiPAP" (Respironics), variable positive airway pressure (VPAP) (ResMed), bilevel positive airway pressure, noninvasive ventilation; Device: noninvasive positive pressure ventilation; Each subject will undergo 2 hours of low-level noninvasive positive pressure ventilation, with an inspiratory positive airway pressure of 6 cm H2O and an expiratory positive airway pressure of 3 cm H2O.; Other Names: BiPAP (Respironics), VPAP (ResMed), bilevel positive airway pressure, noninvasive ventilation
Active Comparator: Low-level ventilation; Each subject will receive 2 hours of low-level noninvasive positive pressure ventilation.	Device: noninvasive positive pressure ventilation; Each subject will undergo 2 hours of high-level noninvasive positive pressure ventilation, with an inspiratory positive airway pressure of 12 cm H2O and an expiratory positive airway pressure of 3 cm H2O.; Other Names: "BiPAP" (Respironics), variable positive airway pressure (VPAP) (ResMed), bilevel positive airway pressure, noninvasive ventilation; Device: noninvasive positive pressure ventilation; Each subject will undergo 2 hours of low-level noninvasive positive pressure ventilation, with an inspiratory positive airway pressure of 6 cm H2O and an expiratory positive airway pressure of 3 cm H2O.; Other Names: BiPAP (Respironics), VPAP (ResMed), bilevel positive airway pressure, noninvasive ventilation

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Difference in Pulmonary Function and Respiratory Muscle Pressure Testing	5 hours
Difference in Gas Exchange	5 hours
Difference in Subjective Dyspnea Between Baseline and the Two Different Ventilator Modes	5 hours

Collaborators and Investigators

• Sponsor: Columbia University
• Investigators: Principal Investigator: Robert C Basner, MD, Columbia University

Study record dates

Study Major Dates

• Study Start: September 1, 2007
• Primary Completion (Actual): August 1, 2012
• Study Completion (Actual): August 1, 2012

Study Registration Dates

• First Submitted: September 27, 2007
• First Submitted That Met QC Criteria: September 27, 2007
• First Posted (Estimate): October 1, 2007

Study Record Updates

• Last Update Posted (Actual): November 27, 2020
• Last Update Submitted That Met QC Criteria: November 5, 2020
• Last Verified: November 1, 2020

More Information

Terms related to this study

• Keywords: noninvasive ventilation
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: AAAC7394