Early Treatment of Amyotrophic Lateral Sclerosis (ALS) With Nutrition and Non-Invasive Positive Pressure Ventilation
Condition: Amyotrophic Lateral Sclerosis
MeSH terms: Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis
Study type: Interventional

Summary: 
      The purposes of the study are to determine the energy balance and evaluate the nutritional
      status of patients with ALS, and to investigate the use of NIPPV as respiratory support to
      treat patients with ALS.
    

Detailed description: 
      Studies to date indicate that percutaneous endoscopic gastrostomy (PEG)-insertion of a
      feeding tube in the stomach-and non-invasive positive pressure ventilation
      (NIPPV)-mechanically assisted or generated breaths delivered through a tightly fitting nasal
      or facial mask-improve survival in amyotrophic lateral sclerosis (ALS), even when introduced
      late in the disease.

      Dr. Kasarskis and his research team believe early intervention with these treatments may
      improve patients outcome even further. However, many issues regarding the early use of these
      treatments prevent the design of a phase III clinical trial to test this hypothesis. Common
      to both NIPPV and nutrition is a lack of a reliable indicator of early respiratory or
      nutritional insufficiency. With respect to nutrition, reliable and cost effective methods are
      needed to determine a patient's energy (i.e. caloric) requirements at different stages of the
      illness to establish a basis for recommending PEG on the adequacy of energy intake. For
      NIPPV, factors that influence acceptance and tolerability of this therapy, and measurements
      of early respiratory dysfunction need to be identified.

      The purposes of this trial are to develop and validate strategies to improve tolerability of
      NIPPV, identify factors that influence acceptance of NIPPV, and evaluate measures of early
      respiratory failure, other than percentage of forced vital capacity (FVC). Researchers will
      also develop and validate methods to determine energy balance in and evaluate the nutritional
      status of patients with ALS that will be applicable to a multi-center phase III study of
      nutrition and NIPPV.

      The study will be conducted at 11 other sites across the country. Some study sites will focus
      on the nutritional aspects of the trial while the other sites will focus on NIPPV treatment.
      A total of 220 patients will be studied over 2 years.

      Enrollment will end on June 30, 2007. The last patient was followed clinically until June 30,
      2008. The study remained open for sample analysis, data analysis, and assessment of vital
      status until the completion of funding on November 30, 2009.
    

Intervention name(s): Early NIPPV; Standard NIPPV
Intervention type(s): Device; Device
Intervention description(s): Early intervention with non-invasive positive pressure ventilation (NIPPV) at 80% forced vital capacity (FVC).; Standard of care non-invasive positive pressure ventilation (NIPPV) at 50% forced vital capacity (FVC).
Other intervention name(s): BiPAP (Bilevel Positive Airway Pressure); BiPAP (Bilevel Positive Airway Pressure)
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Supportive Care
Keywords: amyotrophic lateral sclerosis; ALS; Lou Gehrig's disease; nutrition; NIPPV; non-invasive positive pressure ventilation; percutaneous endoscopic gastrostomy; PEG