Overview

This trial is active, not recruiting.

Condition amyotrophic lateral sclerosis (als)
Treatments sleep study-guided adjustment of niv, standard initiation of niv
Sponsor Columbia University
Collaborator ALS Association
Start date February 2008
End date January 2011
Trial size 40 participants
Trial identifier NCT01363882, AAAC6753

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification efficacy study
Intervention model parallel assignment
Masking double blind (subject, investigator)
Primary purpose supportive care
Arm
(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.
standard initiation of niv
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.
(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.
sleep study-guided adjustment of niv
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.

Primary Outcomes

Measure
Change from baseline in spirometric, respiratory muscle strength, and gas exchange measures
time frame: Up to 6 months after starting NIV

Secondary Outcomes

Measure
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

Eligibility Criteria

Male or female participants from 18 years up to 80 years old.

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

Additional Information

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
Principal investigator Robert C Basner, MD
Trial information was received from ClinicalTrials.gov and was last updated in May 2011.
Information provided to ClinicalTrials.gov by Columbia University.