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.

Condition	Intervention
Amyotrophic Lateral Sclerosis	Procedure: non-invasive positive pressure ventilation

Further Study Details: 

Expected Total Enrollment:  220

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.

Ages Eligible for Study:  18 Years   -   80 Years,  Genders Eligible for Study:  Both

Criteria

Inclusion Criteria:

• Willing and able to give informed consent.
• If women of childbearing age, must be non-lactating and surgically sterile or using an effective method of birth control (double-barrier or oral contraception) and have a negative pregnancy test.
• Minorities: All races and ethnic backgrounds.
• Disease inclusion: Clinically possible/definite/probable, or laboratory supported probable, sporadic or familial ALS according to the revised Escorial criteria.
• Best-sitting FVC between 50% and 95% of predicted normal.
• Onset of progressive weakness within 36 months prior to study.

Exclusion Criteria:

• Untreated or unstable hyper or hypothyroidism, untreated or unstable asthma, unstable angina, advanced liver or renal disease, advanced cancer, untreated diabetes or chronic obstructive pulmonary disease (COPD).
• Diagnosis of other motor neuron or other neurological disorder that mimics ALS.
• Diagnosis of other neurodegenerative disease (i.e., Parkinson’s disease, Alzheimer’s disease, etc.)
• Inflammatory bowel disease or malabsorption syndrome.
• Use of NIPPV, mechanical ventilation, or tracheostomy at the time of consent.
• Inability to adhere to study visit schedule or lack of reliable caretaker if participant requires one.
• Pregnant or lactating woman.

Please refer to this study by ClinicalTrials.gov identifier  NCT00116558

Megan Thompson      (859) 323-8509    megant@uky.edu

Colorado
      University of Colorado, Denver,  Colorado,  80262,  United States; Recruiting
Florida
      University of Miami, Miami,  Florida,  33136,  United States; Recruiting
Kentucky
      University of Kentucky, Lexington,  Kentucky,  40536,  United States; Recruiting
New York
      Columbia University, New York,  New York,  10032,  United States; Recruiting
      Beth Israel, New York,  New York,  10003,  United States; Recruiting
      SUNY, Syracuse,  New York,  13210,  United States; Recruiting
Pennsylvania
      Pennsylvania State University, Hershey,  Pennsylvania,  17033,  United States; Recruiting
      Drexel University, Philadelphia,  Pennsylvania,  19103,  United States; Recruiting
Texas
      University of Texas-San Antonio, San Antonio,  Texas,  78229,  United States; Recruiting
Utah
      University of Utah, Salt Lake City,  Utah,  84132,  United States; Recruiting
Vermont
      University of Vermont, Burlington,  Vermont,  54505,  United States; Recruiting

Study chairs or principal investigators

Edward Kasarskis, MD, PhD,  Principal Investigator,  University of Kentucky   

Study ID Numbers:  R01NS045087
Record last reviewed:  June 2005
Last Updated:  June 30, 2005
Record first received:  June 29, 2005
ClinicalTrials.gov Identifier:  NCT00116558
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-07-05