NCT00580593

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
60

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Apr 2007

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2007

Completed
9 months until next milestone

First Submitted

Initial submission to the registry

December 20, 2007

Completed
4 days until next milestone

First Posted

Study publicly available on registry

December 24, 2007

Completed
4.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2012

Completed
Last Updated

July 10, 2013

Status Verified

July 1, 2013

Enrollment Period

4.9 years

First QC Date

December 20, 2007

Last Update Submit

July 9, 2013

Conditions

Amyotrophic Lateral Sclerosis

Keywords

amyotrophic lateral sclerosisALSnocturnal noninvasive positive pressure ventilationNIPPVforced vital capacityFVC

Outcome Measures

Primary Outcomes (1)

  • 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.

Secondary Outcomes (5)

  • The SF-36 will be measured as a measure of quality of life.

    at baseline and every three months

  • Pulmonary function tests including FVC will be measured.

    at baseline and every three months

  • The ALS FRS will be used as a measure of functional outcome.

    at baseline and every three months

  • The BDI/TDI (baseline and transition dyspnea indexes).

    at baseline and every three months

  • 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

Study Arms (2)

1

ACTIVE COMPARATOR
Device: BiPAP® S/T System

2

SHAM COMPARATOR
Other: sham-NIPPV

Interventions

BiPAP® S/T SystemDEVICE

The BiPAP® S/T System is a NIPPV device that provides intermittent ventilatory assistance to people with difficulty breathing secondary to respiratory muscle weakness.

Also known as: Noninvasive Positive Pressure Ventilation (NIPPV)
1
sham-NIPPVOTHER

a sham-device

Also known as: placebo
2

Eligibility Criteria

Age18 Years - 79 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Related Publications (1)

  • Jacobs TL, Brown DL, Baek J, Migda EM, Funckes T, Gruis KL. Trial of early noninvasive ventilation for ALS: A pilot placebo-controlled study. Neurology. 2016 Nov 1;87(18):1878-1883. doi: 10.1212/WNL.0000000000003158. Epub 2016 Aug 31.

    PMID: 27581221DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Kirsten Gruis, MD, MS

    University of Michigan

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Associate Professor

Study Record Dates

First Submitted

December 20, 2007

First Posted

December 24, 2007

Study Start

April 1, 2007

Primary Completion

March 1, 2012

Study Completion

March 1, 2012

Last Updated

July 10, 2013

Record last verified: 2013-07

Locations

US(1)