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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified January 2008 by National Institute of Neurological Disorders and Stroke (NINDS).
Recruitment status was  Active, not recruiting
Sponsor:
Collaborator:
Information provided by:
National Institute of Neurological Disorders and Stroke (NINDS)
ClinicalTrials.gov Identifier:
NCT00116558
First received: June 29, 2005
Last updated: January 8, 2008
Last verified: January 2008
  Purpose

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
Device: BiPAP

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Early Treatment of Amyotrophic Lateral Sclerosis With Nutrition and Non-Invasive Positive Pressure Ventilation

Resource links provided by NLM:


Further study details as provided by National Institute of Neurological Disorders and Stroke (NINDS):

Primary Outcome Measures:
  • ID sensitive early respiratory failure measure; ID NIPPV acceptance & tolerance predictors & develop compliance policy; Validate respiratory questionnaires. [ Time Frame: duration of the trial ] [ Designated as safety issue: No ]
  • Guidelines to standardize NIPPV and nutritional interventions during the conduct of multicenter clinical drug studies. [ Time Frame: duration of the trial ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Develop acceptance & compliance statistics for Phase 3; Estimate survival increase with early NIPPV use for Phase 3 [ Time Frame: duration of the trial ] [ Designated as safety issue: No ]
  • Develop equations to accurately predict TDEE based on clinical measures that are validated against the DLW method. [ Time Frame: duration of the trial ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]

Estimated Enrollment: 220
Study Start Date: August 2004
Estimated Study Completion Date: October 2008
Estimated Primary Completion Date: July 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: intervention group
NIPPV
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

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.

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  • 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00116558

Locations
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
Columbia University
New York, New York, United States, 10032
Beth Israel
New York, New York, United States, 10003
SUNY
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
Investigators
Principal Investigator: Edward Kasarskis, MD, PhD University of Kentucky
  More Information

Additional Information:
No publications provided by National Institute of Neurological Disorders and Stroke (NINDS)

Additional 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
Study First Received: June 29, 2005
Last Updated: January 8, 2008
Health Authority: United States: Federal Government

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

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

ClinicalTrials.gov processed this record on November 25, 2014