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

NCT00116558 | Completed Results DMC FDA Device

• 1 other identifier: R01NS045087
• Study Type: interventional
• Target: 153
• Locations: 1 country
• Sites: 12

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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

amyotrophic lateral sclerosis; ALS; Lou Gehrig's disease; nutrition; NIPPV; non-invasive positive pressure ventilation; percutaneous endoscopic gastrostomy; PEG

Outcome Measures

Primary Outcomes (1)

• 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.

  6 weeks

Other Outcomes (4)

• Patient Survival With Early Versus Standard of Care NIPPV Treatment

  one year

• Compliance With NIPPV Treatment

  one month

• Total Daily Energy Expediture (TDEE) of ALS Patients

  Duration of study (approximately 1 year)

Study Arms (3)

Early NIPPV Intervention

EXPERIMENTAL

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

Device: Early NIPPV

Standard of Care NIPPV

ACTIVE COMPARATOR

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

Device: Standard NIPPV

Standard of Care NIPPV and Nutritional Monitoring

ACTIVE COMPARATOR

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.

Device: Standard NIPPV

Interventions

Early NIPPV DEVICE

Early intervention with non-invasive positive pressure ventilation (NIPPV) at 80% forced vital capacity (FVC).

Also known as: BiPAP (Bilevel Positive Airway Pressure)

Early NIPPV Intervention

Standard NIPPV DEVICE

Standard of care non-invasive positive pressure ventilation (NIPPV) at 50% forced vital capacity (FVC).

Also known as: BiPAP (Bilevel Positive Airway Pressure)

Standard of Care NIPPV; Standard of Care NIPPV and Nutritional Monitoring

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Edward Kasaraskis: lead
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator

Study Sites (12)

University of Colorado

Denver, Colorado, 80262, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

University of Kentucky

Lexington, Kentucky, 40536, United States

Location

Henry Ford Hospital

Detroit, Michigan, 48202, United States

Location

Beth Israel

New York, New York, 10003, United States

Location

Columbia University

New York, New York, 10032, United States

Location

SUNY

Syracuse, New York, 13210, United States

Location

Pennsylvania State University

Hershey, Pennsylvania, 17033, United States

Location

Drexel University

Philadelphia, Pennsylvania, 19103, United States

Location

University of Texas-San Antonio

San Antonio, Texas, 78229, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

University of Vermont

Burlington, Vermont, 54505, United States

Location

Related Publications (3)

• 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.

  PMID: 24522445 BACKGROUND

• 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.

  PMID: 29243507 BACKGROUND

• 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.

  PMID: 21271789 RESULT

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Continuous Positive Airway Pressure

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Positive-Pressure Respiration; Respiration, Artificial; Airway Management; Therapeutics; Respiratory Therapy

Limitations and Caveats

Compliance with NIPPV intervention is limited due to technical difficulties in obtaining BiPAP download data from some sites. This was due to variability in the local respiratory therapists engaged by commercial respiratory therapy companies.

Results Point of Contact

• Title: Edward J. Kasarskis, M.D., Ph.D.
• Organization: University of Kentucky

ejkas@uky.edu

859-218-5061

Study Officials

• Edward Kasarskis, MD, PhD

  University of Kentucky

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: June 29, 2005
• First Posted: June 30, 2005
• Study Start: August 1, 2004
• Primary Completion: January 1, 2012
• Study Completion: January 1, 2012
• Last Updated: July 31, 2019
• Results First Posted: July 31, 2019

Record last verified: 2019-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (12)