Clinical trial NCT00386464: Noninvasive Ventilation in ALS Patients With Mild Respiratory Involvement
Official title: Noninvasive Ventilation in ALS Patients With Mild Respiratory Involvement
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Amyotrophic Lateral Sclerosis
Keywords: bipap
lou gehrigs disease
motor neuron disease
respiratory muscles
Lead sponsor: Johns Hopkins University
Sponsor class: OTHER
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
First posted: 2006-10-11
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: SUPPORTIVE_CARE

Brief summary: Non-invasive ventilation or BiPAP®, which is a form of breathing support delivered through a facemask, is a successful treatment for the respiratory complications of amyotrophic lateral sclerosis (ALS). It has been shown to prolong survival, improve quality of life, and improve cognitive function. It is widely used among patients with ALS who have advanced breathing difficulties. It is not known whether there is benefit to using non-invasive ventilation earlier in the disease course.

There is evidence that non-invasive ventilation may slow down the decline in breathing function. If this were true then it would make sense to start non-invasive ventilation use earlier than the current clinically accepted practices.

The purpose of this study is to determine whether using non-invasive ventilation early in the course of disease can slow the decline in breathing function.

Patients remain in the study for 6 months and are asked to make 7 clinic visits during which time they will undergo pulmonary function tests and complete questionnaires.

Detailed description: This is a randomized, crossover trial for patient with ALS and mild respiratory involvement. Patients with forced vital capacity above 60% of the predicted value can join. Patients will be assigned to either start using non-invasive ventilation at night or continue their usual care. After three months, patients will switch over to the other treatment group. For example, a patient who was initially assigned to continue their usual care would begin using non-invasive ventilation after three months.

Eligibility criteria: Inclusion Criteria:

* Probable or definite ALS by El Escorial criteria
* age \>17 years
* FVC \>60
* minimal respiratory symptoms (no orthopnea or dyspnea at rest)
* ability to provide informed consent

Exclusion Criteria:

* Presence of another neurodegenerative disease
* arterial CO2 above 45 mmHg
* O2 below 60 mmHg
* coexisting chronic lung disease unrelated to ALS
* presence of an unstable medical condition such as coronary artery disease, liver failure, renal failure or cancer in the 30 days preceding enrollment

Primary outcomes:
measure: Decline in forced vital capacity
Secondary outcomes:
measure: quality of life
measure: respiratory quality of life
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Johns Hopkins University School of Medicine
state: Maryland
Study officials:
affiliation: Johns Hopkins University
name: Noah Lechtzin, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Johns Hopkins University
name: Charles M Wiener, MD
role: STUDY_CHAIR
References: