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

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

Pilot Placebo-Controlled Trial of Early Noninvasive Ventilation for ALS

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00580593
• Other study ID #: R01NS55200
• Status: Completed
• Phase: Phase 1
• First received: December 20, 2007
• Last updated: July 9, 2013
• Start date: April 2007
• Est. completion date: March 2012

Study information

• Verified date: July 2013
• Source: University of Michigan
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal GovernmentUnited States: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 60
• Est. completion date: March 2012
• Est. primary completion date: March 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 79 Years

Eligibility

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

Study Design

Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

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:
• sham-NIPPV
a sham-device

Locations

Country	Name	City	State
United States	University of Michigan	Ann Arbor	Michigan

Sponsors (2)

Lead Sponsor	Collaborator
University of Michigan	National Institute of Neurological Disorders and Stroke (NINDS)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.		every three months, for the duration of the disease or as long as the person with ALS is able to participate in the study.	No
Secondary	The SF-36 will be measured as a measure of quality of life.		at baseline and every three months	No
Secondary	Pulmonary function tests including FVC will be measured.		at baseline and every three months	No
Secondary	The ALS FRS will be used as a measure of functional outcome.		at baseline and every three months	No
Secondary	The BDI/TDI (baseline and transition dyspnea indexes).		at baseline and every three months	No
Secondary	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.		at baseline and every three months	No