Trial of Early Noninvasive Ventilation for Amyotrophic Lateral Sclerosis (ALS)

NCT ID: NCT00580593

Last Updated: 2013-07-10

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 60 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-04-30
• Study Completion Date: 2012-03-31

Brief Summary

The goal of this trial is to determine the feasibility of conducting a randomized, double-blind, placebo-controlled trial of nocturnal noninvasive positive pressure ventilation in persons with amyotrophic lateral sclerosis with an forced vital capacity greater than or equal to 50 percent.

Detailed Description

Amyotrophic lateral sclerosis (ALS) is an untreatable neurodegenerative disorder characterized by the progressive loss of motor neuron function. Respiratory failure is the most common cause of death in persons with ALS. Treatment of respiratory muscle weakness with nocturnal noninvasive positive pressure ventilation (NIPPV) when forced vital capacity (FVC) is less than 50 percent-but prior to the development of respiratory failure-has prolonged survival in observational studies. Despite the association of NIPPV use and survival, it is unknown whether earlier NIPPV treatment will benefit people with ALS. Also, no placebo-controlled studies of NIPPV treatment and people with ALS have been conducted.

The goals of the this trial are to assess the feasibility of conducting a prospective, randomized, double-blind, clinical trial of NIPPV versus control (sham) NIPPV in people with ALS who have a FVC greater than 50 percent, and to gain preliminary data on outcome effects.

In the trial, the investigators will test the following hypotheses: (1) People with ALS who have a FVC greater than 50 percent can tolerate active NIPPV and control NIPPV, and will find control NIPPV to be a believable treatment; (2) Initiation of active NIPPV in people with ALS who have a FVC greater than 50 percent will have better clinical outcomes with respect to measures of quality of life, rate of pulmonary function decline, and functional outcome; and (3) People with ALS who start active NIPPV early will have improved tolerance later when respiratory weakness has progressed. These aims will enable planning of a subsequent, large-scale and definitive clinical trial of early NIPPV (FVC greater than 50 percent) in people with ALS.

If the benefits of early NIPPV can be confirmed, then a new treatment may be established for this progressive, fatal disease.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

1

Group Type: ACTIVE_COMPARATOR

BiPAP® S/T System

Intervention Type: DEVICE

The BiPAP® S/T System is a NIPPV device that provides intermittent ventilatory assistance to people with difficulty breathing secondary to respiratory muscle weakness.

2

Group Type: SHAM_COMPARATOR

sham-NIPPV

Intervention Type: OTHER

a sham-device

Interventions

BiPAP® S/T System

The BiPAP® S/T System is a NIPPV device that provides intermittent ventilatory assistance to people with difficulty breathing secondary to respiratory muscle weakness.

Intervention Type: DEVICE

sham-NIPPV

a sham-device

Intervention Type: OTHER

Other Intervention Names

Noninvasive Positive Pressure Ventilation (NIPPV); placebo

Eligibility Criteria

Inclusion Criteria

• Age > 18 years old
• Probable or possible ALS by El Escorial criteria
• If of child-bearing potential, has a negative urine or serum pregnancy test

Exclusion Criteria

• FVC < 50% predicted for age
• Previous pneumothorax
• Bullous emphysema
• Requirement for oxygen
• Previous use of any positive pressure ventilation equipment (continuous positive airway pressure, or bilevel positive airway pressure)
• Current involvement in a clinical treatment trial
• Any unstable medical condition thought likely to interfere with participation

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 79 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Institute of Neurological Disorders and Stroke (NINDS)

NIH

Sponsor Role: collaborator

University of Michigan

OTHER

Sponsor Role: lead

Responsible Party

Kirsten Gruis

Associate Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Kirsten Gruis, MD, MS

Role: PRINCIPAL_INVESTIGATOR

University of Michigan

Locations

University of Michigan

Ann Arbor, Michigan, United States

Countries

United States

References

Jacobs TL, Brown DL, Baek J, Migda EM, Funckes T, Gruis KL. Trial of early noninvasive ventilation for ALS: A pilot placebo-controlled study. Neurology. 2016 Nov 1;87(18):1878-1883. doi: 10.1212/WNL.0000000000003158. Epub 2016 Aug 31.

Reference Type: DERIVED

PMID: 27581221 (View on PubMed)

Other Identifiers

R01NS55200

Identifier Type: -

Identifier Source: org_study_id