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Early Treatment of Amyotrophic Lateral Sclerosis (ALS) With Nutrition and Non-Invasive Positive Pressure Ventilation (NIPPV)

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ClinicalTrials.gov Identifier: NCT00116558
Recruitment Status : Unknown
Verified January 2008 by National Institute of Neurological Disorders and Stroke (NINDS).
Recruitment status was:  Active, not recruiting
First Posted : June 30, 2005
Last Update Posted : January 15, 2008
Information provided by:

Study Description
Brief 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.

Condition or disease Intervention/treatment
Amyotrophic Lateral Sclerosis Device: BiPAP

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.

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 220 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Early Treatment of Amyotrophic Lateral Sclerosis With Nutrition and Non-Invasive Positive Pressure Ventilation
Study Start Date : August 2004
Estimated Primary Completion Date : July 2008
Estimated Study Completion Date : October 2008

Arms and Interventions

Arm Intervention/treatment
Experimental: intervention group
Device: BiPAP
non-invasive positive pressure ventilation (NIPPV) - Early Intervention with NIPPV NIPPV involves mechanically assisted or generated breaths delivered through a tightly fitting nasal or facial mask
No Intervention: Nutrition

Outcome Measures

Primary Outcome Measures :
  1. ID sensitive early respiratory failure measure; ID NIPPV acceptance & tolerance predictors & develop compliance policy; Validate respiratory questionnaires. [ Time Frame: duration of the trial ]
  2. Guidelines to standardize NIPPV and nutritional interventions during the conduct of multicenter clinical drug studies. [ Time Frame: duration of the trial ]

Secondary Outcome Measures :
  1. Develop acceptance & compliance statistics for Phase 3; Estimate survival increase with early NIPPV use for Phase 3 [ Time Frame: duration of the trial ]
  2. Develop equations to accurately predict TDEE based on clinical measures that are validated against the DLW method. [ Time Frame: duration of the trial ]
  3. Identify optimal method to measure Energy Intake (EI). Formulate criteria for intervention with PEG based on maintaining energy balance [ Time Frame: duration of the trial ]

Eligibility Criteria

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.
  • Onset of progressive weakness within 60 months prior to study.
  • Willing to return for visits as scheduled and adhere to protocol requirements.

FVC Criteria

  • NIPPV Arm: Best-sitting FVC between 50% and 95% of predicted normal.
  • Nutrition Arm: Best-sitting FVC >50% of predicted normal.

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.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00116558

United States, Colorado
University of Colorado
Denver, Colorado, United States, 80262
United States, Florida
University of Miami
Miami, Florida, United States, 33136
United States, Kentucky
University of Kentucky
Lexington, Kentucky, United States, 40536
United States, Michigan
Henry Ford Hospital
Detroit, Michigan, United States, 48202
United States, New York
Beth Israel
New York, New York, United States, 10003
Columbia University
New York, New York, United States, 10032
Syracuse, New York, United States, 13210
United States, Pennsylvania
Pennsylvania State University
Hershey, Pennsylvania, United States, 17033
Drexel University
Philadelphia, Pennsylvania, United States, 19103
United States, Texas
University of Texas-San Antonio
San Antonio, Texas, United States, 78229
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84132
United States, Vermont
University of Vermont
Burlington, Vermont, United States, 54505
Sponsors and Collaborators
University of Kentucky
National Institute of Neurological Disorders and Stroke (NINDS)
Principal Investigator: Edward Kasarskis, MD, PhD University of Kentucky
More Information

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Edward Kasarskis, MD, PhD ,, University of Kentucky
ClinicalTrials.gov Identifier: NCT00116558     History of Changes
Other Study ID Numbers: R01NS045087 ( U.S. NIH Grant/Contract )
First Posted: June 30, 2005    Key Record Dates
Last Update Posted: January 15, 2008
Last Verified: January 2008

Keywords provided by National Institute of Neurological Disorders and Stroke (NINDS):
amyotrophic lateral sclerosis
Lou Gehrig's disease
non-invasive positive pressure ventilation
percutaneous endoscopic gastrostomy

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases