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

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ClinicalTrials.gov Identifier: NCT00116558
Recruitment Status : Completed
First Posted : June 30, 2005
Results First Posted : July 31, 2019
Last Update Posted : July 31, 2019
Sponsor:
Collaborator:
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):
Edward Kasaraskis, University of Kentucky

Tracking Information
First Submitted Date  ICMJE June 29, 2005
First Posted Date  ICMJE June 30, 2005
Results First Submitted Date  ICMJE March 15, 2010
Results First Posted Date  ICMJE July 31, 2019
Last Update Posted Date July 31, 2019
Actual Study Start Date  ICMJE August 1, 2004
Actual Primary Completion Date January 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 11, 2019)
Acceptance Rate of Early Non-invasive Positive Pressure Ventilation (NIPPV) Treatment. [ Time Frame: 6 weeks ]
Percentage of patients attempting to use NIPPV therapy within six weeks of initial offer.
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00116558 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures
 (submitted: July 11, 2019)
  • Patient Survival With Early Versus Standard of Care NIPPV Treatment [ Time Frame: one year ]
    Duration of patient survival
  • Compliance With NIPPV Treatment [ Time Frame: one month ]
    Number of hours of NIPPV use per month
  • Total Daily Energy Expediture (TDEE) of ALS Patients [ Time Frame: Duration of study (approximately 1 year) ]
    Total daily energy expenditure (TDEE) with be measured using the dual labeled water (DLW) method
  • Tolerance of NIPPV Treatment [ Time Frame: one month ]
    Percent of patients achieving tolerance (>4hr/night NIPPV after 2-3 week titration period) determined by actual usage data.
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Early Treatment of Amyotrophic Lateral Sclerosis (ALS) With Nutrition and Non-Invasive Positive Pressure Ventilation
Official Title  ICMJE Early Treatment of Amyotrophic Lateral Sclerosis With Nutrition and Non-Invasive Positive Pressure Ventilation
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.
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
Condition  ICMJE Amyotrophic Lateral Sclerosis
Intervention  ICMJE
  • Device: Early NIPPV
    Early intervention with non-invasive positive pressure ventilation (NIPPV) at 80% forced vital capacity (FVC).
    Other Name: BiPAP (Bilevel Positive Airway Pressure)
  • Device: Standard NIPPV
    Standard of care non-invasive positive pressure ventilation (NIPPV) at 50% forced vital capacity (FVC).
    Other Name: BiPAP (Bilevel Positive Airway Pressure)
Study Arms  ICMJE
  • Experimental: Early NIPPV Intervention
    Participants with >80% predicted forced vital capacity (FVC).
    Intervention: Device: Early NIPPV
  • Active Comparator: Standard of Care NIPPV
    Participants with 50-74% predicted forced vital capacity (FVC).
    Intervention: Device: Standard NIPPV
  • Active Comparator: Standard of Care NIPPV and Nutritional Monitoring
    Participants with 50-95% forced vital capacity (FVC), and normal or impaired amyotrophic lateral sclerosis functional rating scale (ALSFRS) scores. Participants in this group will receive standard of care NIPPV therapy but will also undergo detailed analysis of nutritional status.
    Intervention: Device: Standard NIPPV
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 11, 2019)
153
Original Enrollment  ICMJE
 (submitted: June 29, 2005)
220
Actual Study Completion Date  ICMJE January 2012
Actual Primary Completion Date January 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00116558
Other Study ID Numbers  ICMJE R01NS045087( U.S. NIH Grant/Contract )
R01NS045087 ( U.S. NIH Grant/Contract )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Edward Kasaraskis, University of Kentucky
Study Sponsor  ICMJE Edward Kasaraskis
Collaborators  ICMJE National Institute of Neurological Disorders and Stroke (NINDS)
Investigators  ICMJE
Principal Investigator: Edward Kasarskis, MD, PhD University of Kentucky
PRS Account University of Kentucky
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP