Trial of Early Noninvasive Ventilation for Amyotrophic Lateral Sclerosis (ALS)

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Kirsten Gruis, University of Michigan
ClinicalTrials.gov Identifier:
NCT00580593
First received: December 20, 2007
Last updated: July 9, 2013
Last verified: July 2013
  Purpose

The goal of this trial is to determine the feasibility of conducting a randomized, double-blind, placebo-controlled trial of nocturnal noninvasive positive pressure ventilation in persons with amyotrophic lateral sclerosis with an forced vital capacity greater than or equal to 50 percent.


Condition Intervention Phase
Amyotrophic Lateral Sclerosis
Device: BiPAP® S/T System
Other: sham-NIPPV
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Pilot Placebo-Controlled Trial of Early Noninvasive Ventilation for ALS

Resource links provided by NLM:


Further study details as provided by University of Michigan:

Primary Outcome Measures:
  • NIPPV adherence, as summarized by weekly means and standard deviations of hours of use for the two groups (active-NIPPV and sham-NIPPV). Additionally the patients' belief as to which treatment they believed they had tried will be assessed. [ Time Frame: every three months, for the duration of the disease or as long as the person with ALS is able to participate in the study. ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • The SF-36 will be measured as a measure of quality of life. [ Time Frame: at baseline and every three months ] [ Designated as safety issue: No ]
  • Pulmonary function tests including FVC will be measured. [ Time Frame: at baseline and every three months ] [ Designated as safety issue: No ]
  • The ALS FRS will be used as a measure of functional outcome. [ Time Frame: at baseline and every three months ] [ Designated as safety issue: No ]
  • The BDI/TDI (baseline and transition dyspnea indexes). [ Time Frame: at baseline and every three months ] [ Designated as safety issue: No ]
  • Tolerance to standard NIPPV treatment during the observational phase of the study (after FVC has fallen below 50%) will also be summarized by weekly means and standard deviations of hours of use. [ Time Frame: at baseline and every three months ] [ Designated as safety issue: No ]

Enrollment: 60
Study Start Date: April 2007
Study Completion Date: March 2012
Primary Completion Date: March 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: 1 Device: BiPAP® S/T System
The BiPAP® S/T System is a NIPPV device that provides intermittent ventilatory assistance to people with difficulty breathing secondary to respiratory muscle weakness.
Other Name: Noninvasive Positive Pressure Ventilation (NIPPV)
Sham Comparator: 2 Other: sham-NIPPV
a sham-device
Other Name: placebo

Detailed Description:

Amyotrophic lateral sclerosis (ALS) is an untreatable neurodegenerative disorder characterized by the progressive loss of motor neuron function. Respiratory failure is the most common cause of death in persons with ALS. Treatment of respiratory muscle weakness with nocturnal noninvasive positive pressure ventilation (NIPPV) when forced vital capacity (FVC) is less than 50 percent—but prior to the development of respiratory failure—has prolonged survival in observational studies. Despite the association of NIPPV use and survival, it is unknown whether earlier NIPPV treatment will benefit people with ALS. Also, no placebo-controlled studies of NIPPV treatment and people with ALS have been conducted.

The goals of the this trial are to assess the feasibility of conducting a prospective, randomized, double-blind, clinical trial of NIPPV versus control (sham) NIPPV in people with ALS who have a FVC greater than 50 percent, and to gain preliminary data on outcome effects.

In the trial, the investigators will test the following hypotheses: (1) People with ALS who have a FVC greater than 50 percent can tolerate active NIPPV and control NIPPV, and will find control NIPPV to be a believable treatment; (2) Initiation of active NIPPV in people with ALS who have a FVC greater than 50 percent will have better clinical outcomes with respect to measures of quality of life, rate of pulmonary function decline, and functional outcome; and (3) People with ALS who start active NIPPV early will have improved tolerance later when respiratory weakness has progressed. These aims will enable planning of a subsequent, large-scale and definitive clinical trial of early NIPPV (FVC greater than 50 percent) in people with ALS.

If the benefits of early NIPPV can be confirmed, then a new treatment may be established for this progressive, fatal disease.

  Eligibility

Ages Eligible for Study:   18 Years to 79 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age > 18 years old
  • Probable or possible ALS by El Escorial criteria
  • If of child-bearing potential, has a negative urine or serum pregnancy test

Exclusion Criteria:

  • FVC < 50% predicted for age
  • Previous pneumothorax
  • Bullous emphysema
  • Requirement for oxygen
  • Previous use of any positive pressure ventilation equipment (continuous positive airway pressure, or bilevel positive airway pressure)
  • Current involvement in a clinical treatment trial
  • Any unstable medical condition thought likely to interfere with participation
  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: NCT00580593

Locations
United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
Sponsors and Collaborators
University of Michigan
Investigators
Principal Investigator: Kirsten Gruis, MD, MS University of Michigan
  More Information

No publications provided

Responsible Party: Kirsten Gruis, Associate Professor, University of Michigan
ClinicalTrials.gov Identifier: NCT00580593     History of Changes
Other Study ID Numbers: R01NS55200
Study First Received: December 20, 2007
Last Updated: July 9, 2013
Health Authority: United States: Federal Government
United States: Institutional Review Board

Keywords provided by University of Michigan:
amyotrophic lateral sclerosis
ALS
nocturnal noninvasive positive pressure ventilation
NIPPV
forced vital capacity
FVC

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

ClinicalTrials.gov processed this record on July 20, 2014