Trial of Early Noninvasive Ventilation for Amyotrophic Lateral Sclerosis (ALS)
Condition: Amyotrophic Lateral Sclerosis
MeSH terms: Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis
Study type: Interventional
Phase: Phase 1

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.
    

Intervention name(s): BiPAP® S/T System; sham-NIPPV
Intervention type(s): Device; Other
Intervention description(s): The BiPAP® S/T System is a NIPPV device that provides intermittent ventilatory assistance to people with difficulty breathing secondary to respiratory muscle weakness.; a sham-device
Other intervention name(s): Noninvasive Positive Pressure Ventilation (NIPPV); placebo
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: amyotrophic lateral sclerosis; ALS; nocturnal noninvasive positive pressure ventilation; NIPPV; forced vital capacity; FVC