Polysomnography-directed Noninvasive Ventilation in Amyotrophic Lateral Sclerosis (ALS)

NCT01363882 | Terminated

• 1 other identifier: AAAC6753
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

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.

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Keywords

Amyotrophic Lateral Sclerosis; Noninvasive Ventilation; Respiratory Dysfunction; Positive Pressure Ventilation

Outcome Measures

Primary Outcomes (1)

• 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

  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)

  Up to 6 months after starting NIV

• Survival

  Up to 6 months after baseline

• Nocturnal oxygenation and ventilation

  Up to 6 months after starting NIV

• Modified Borg dyspnea score (see description)

  Up to 6 months after baseline

Study Arms (2)

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.

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.

Other: Sleep study-guided adjustment of NIV

Interventions

Sleep study-guided adjustment of NIV OTHER

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.

Sleep study titrated NIV

Standard initiation of NIV OTHER

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.

Standard NIV

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of probable or definite Amyotrophic Lateral Sclerosis (ALS) per El Escorial criteria
• Between ages of 18 and 80 yrs old

You may not qualify if:

• 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

Sponsors & Collaborators

• Columbia University: lead
• ALS Association: collaborator

Study Sites (1)

Columbia University Medical Center

New York, New York, 10032, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Dyspnea

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Respiration Disorders; Respiratory Tract Diseases; Signs and Symptoms, Respiratory; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Robert C Basner, MD

  Columbia University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 28, 2011
• First Posted: June 2, 2011
• Study Start: February 1, 2008
• Primary Completion: July 1, 2010
• Study Completion: July 1, 2010
• Last Updated: November 13, 2020

Record last verified: 2020-11

Locations

US (1)