A Phase 1 interventional study of BiPAP® S/T System and sham-NIPPV in Amyotrophic Lateral Sclerosis, sponsored by University of Michigan. Completed at 1 site in United States. Open to participants aged 18 Years to 79 Years. Per ClinicalTrials.gov, last updated 2013-07-10.
Sponsored by University of Michigan · Phase 1, Interventional, and Treatment
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.
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.
717 studies on the registry are indexed under Motor Neuron Disease; 137 are open to participants now.
This study's enrollment of 60 is above the median of 35 across 461 interventional studies indexed under Motor Neuron Disease.
Browse Motor Neuron Disease studies →University of Michigan is the lead sponsor of 1,475 studies on the registry; 196 are open to participants now.
Of its 162 completed or terminated interventional studies of FDA-regulated products, 128 (79%) have results posted.
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Exclusion Criteria:
Device: BiPAP® S/T System
Other: sham-NIPPV
The BiPAP® S/T System is a NIPPV device that provides intermittent ventilatory assistance to people with difficulty breathing secondary to respiratory muscle weakness.
Also known as: Noninvasive Positive Pressure Ventilation (NIPPV)
a sham-device
Also known as: placebo
NIPPV adherence, as summarized by weekly means and standard deviations of hours of use for the two groups (active-NIPPV and sham-NIPPV). Additionally the patients' belief as to which treatment they believed they had tried will be assessed.
Time frame: every three months, for the duration of the disease or as long as the person with ALS is able to participate in the study.
The SF-36 will be measured as a measure of quality of life.
Time frame: at baseline and every three months
Pulmonary function tests including FVC will be measured.
Time frame: at baseline and every three months
The ALS FRS will be used as a measure of functional outcome.
Time frame: at baseline and every three months
The BDI/TDI (baseline and transition dyspnea indexes).
Time frame: at baseline and every three months
Tolerance to standard NIPPV treatment during the observational phase of the study (after FVC has fallen below 50%) will also be summarized by weekly means and standard deviations of hours of use.
Time frame: at baseline and every three months
This study is completed, as verified in Jul 2013. You cannot join it, but the record below documents what was studied.
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