search
Back to results

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

Primary Purpose

Amyotrophic Lateral Sclerosis

Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Early NIPPV
Standard NIPPV
Sponsored by
Edward Kasaraskis
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional supportive care trial for Amyotrophic Lateral Sclerosis focused on measuring amyotrophic lateral sclerosis, ALS, Lou Gehrig's disease, nutrition, NIPPV, non-invasive positive pressure ventilation, percutaneous endoscopic gastrostomy, PEG

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • University of Colorado
  • University of Miami
  • University of Kentucky
  • Henry Ford Hospital
  • Beth Israel
  • Columbia University
  • SUNY
  • Pennsylvania State University
  • Drexel University
  • University of Texas-San Antonio
  • University of Utah
  • University of Vermont

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Active Comparator

Active Comparator

Arm Label

Early NIPPV Intervention

Standard of Care NIPPV

Standard of Care NIPPV and Nutritional Monitoring

Arm Description

Participants with >80% predicted forced vital capacity (FVC).

Participants with 50-74% predicted forced vital capacity (FVC).

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.

Outcomes

Primary Outcome Measures

Acceptance Rate of Early Non-invasive Positive Pressure Ventilation (NIPPV) Treatment.
Percentage of patients attempting to use NIPPV therapy within six weeks of initial offer.

Secondary Outcome Measures

Full Information

First Posted
June 29, 2005
Last Updated
July 11, 2019
Sponsor
Edward Kasaraskis
Collaborators
National Institute of Neurological Disorders and Stroke (NINDS)
search

1. Study Identification

Unique Protocol Identification Number
NCT00116558
Brief Title
Early Treatment of Amyotrophic Lateral Sclerosis (ALS) With Nutrition and Non-Invasive Positive Pressure Ventilation
Official Title
Early Treatment of Amyotrophic Lateral Sclerosis With Nutrition and Non-Invasive Positive Pressure Ventilation
Study Type
Interventional

2. Study Status

Record Verification Date
July 2019
Overall Recruitment Status
Completed
Study Start Date
August 1, 2004 (Actual)
Primary Completion Date
January 2012 (Actual)
Study Completion Date
January 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Edward Kasaraskis
Collaborators
National Institute of Neurological Disorders and Stroke (NINDS)

4. Oversight

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
Data Monitoring Committee
Yes

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
Keywords
amyotrophic lateral sclerosis, ALS, Lou Gehrig's disease, nutrition, NIPPV, non-invasive positive pressure ventilation, percutaneous endoscopic gastrostomy, PEG

7. Study Design

Primary Purpose
Supportive Care
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
153 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Early NIPPV Intervention
Arm Type
Experimental
Arm Description
Participants with >80% predicted forced vital capacity (FVC).
Arm Title
Standard of Care NIPPV
Arm Type
Active Comparator
Arm Description
Participants with 50-74% predicted forced vital capacity (FVC).
Arm Title
Standard of Care NIPPV and Nutritional Monitoring
Arm Type
Active Comparator
Arm Description
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 Type
Device
Intervention Name(s)
Early NIPPV
Other Intervention Name(s)
BiPAP (Bilevel Positive Airway Pressure)
Intervention Description
Early intervention with non-invasive positive pressure ventilation (NIPPV) at 80% forced vital capacity (FVC).
Intervention Type
Device
Intervention Name(s)
Standard NIPPV
Other Intervention Name(s)
BiPAP (Bilevel Positive Airway Pressure)
Intervention Description
Standard of care non-invasive positive pressure ventilation (NIPPV) at 50% forced vital capacity (FVC).
Primary Outcome Measure Information:
Title
Acceptance Rate of Early Non-invasive Positive Pressure Ventilation (NIPPV) Treatment.
Description
Percentage of patients attempting to use NIPPV therapy within six weeks of initial offer.
Time Frame
6 weeks
Other Pre-specified Outcome Measures:
Title
Patient Survival With Early Versus Standard of Care NIPPV Treatment
Description
Duration of patient survival
Time Frame
one year
Title
Compliance With NIPPV Treatment
Description
Number of hours of NIPPV use per month
Time Frame
one month
Title
Total Daily Energy Expediture (TDEE) of ALS Patients
Description
Total daily energy expenditure (TDEE) with be measured using the dual labeled water (DLW) method
Time Frame
Duration of study (approximately 1 year)
Title
Tolerance of NIPPV Treatment
Description
Percent of patients achieving tolerance (>4hr/night NIPPV after 2-3 week titration period) determined by actual usage data.
Time Frame
one month

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Edward Kasarskis, MD, PhD
Organizational Affiliation
University of Kentucky
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Colorado
City
Denver
State/Province
Colorado
ZIP/Postal Code
80262
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
University of Kentucky
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40536
Country
United States
Facility Name
Henry Ford Hospital
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48202
Country
United States
Facility Name
Beth Israel
City
New York
State/Province
New York
ZIP/Postal Code
10003
Country
United States
Facility Name
Columbia University
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
SUNY
City
Syracuse
State/Province
New York
ZIP/Postal Code
13210
Country
United States
Facility Name
Pennsylvania State University
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Drexel University
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19103
Country
United States
Facility Name
University of Texas-San Antonio
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
University of Vermont
City
Burlington
State/Province
Vermont
ZIP/Postal Code
54505
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
24522445
Citation
Kasarskis EJ, Mendiondo MS, Matthews DE, Mitsumoto H, Tandan R, Simmons Z, Bromberg MB, Kryscio RJ; ALS Nutrition/NIPPV Study Group. Estimating daily energy expenditure in individuals with amyotrophic lateral sclerosis. Am J Clin Nutr. 2014 Apr;99(4):792-803. doi: 10.3945/ajcn.113.069997. Epub 2014 Feb 12.
Results Reference
background
PubMed Identifier
29243507
Citation
Scagnelli CN, Howard DB, Bromberg MB, Kasarskis EJ, Matthews DE, Mitsumoto HM, Simmons Z, Tandan R; ALS Nutrition-NIPPV Study Group. Hydration measured by doubly labeled water in ALS and its effects on survival. Amyotroph Lateral Scler Frontotemporal Degener. 2018 May;19(3-4):220-231. doi: 10.1080/21678421.2017.1413117. Epub 2017 Dec 15.
Results Reference
background
PubMed Identifier
21271789
Citation
Kasarskis EJ, Mendiondo MS, Wells S, Malguizo MS, Thompson M, Healey M, Kryscio RJ; ALS Nutrition/NIPPV Study Group. The ALS Nutrition/NIPPV Study: design, feasibility, and initial results. Amyotroph Lateral Scler. 2011 Jan;12(1):17-25. doi: 10.3109/17482968.2010.515225.
Results Reference
result

Learn more about this trial

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

We'll reach out to this number within 24 hrs