Effects of Nocturnal Non-invasive Ventilation in Patients With Amyotrophic Lateral Sclerosis

This study has been completed.
Sponsor:
Collaborator:
Taipei Veterans General Hospital, Taiwan
Information provided by (Responsible Party):
National Taiwan University Hospital
ClinicalTrials.gov Identifier:
NCT00958048
First received: February 8, 2009
Last updated: August 14, 2013
Last verified: August 2013
  Purpose

Specific aims:

Aim 1. To determine the incidence of hypoventilation in Amyotrophic Lateral Sclerosis (ALS) patients.

Aim 2. To identify the clinical characteristics and risk factors associated .

Aim 3. To determine the effect of early intervention with nocturnal NIV on the prognosis of ALS patients.


Condition Intervention
Amyotrophic Lateral Sclerosis
Device: non-invasive ventilation

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Effects of Early Intervention With Nocturnal Non-invasive Ventilation in Patients With Amyotrophic Lateral Sclerosis: A Randomized, Controlled Trial

Resource links provided by NLM:


Further study details as provided by National Taiwan University Hospital:

Primary Outcome Measures:
  • Survival [ Time Frame: 36 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Changes of PtcCO2 and PaCO2, admission or unexpected clinical visiting, and QoL [ Time Frame: 36 months ] [ Designated as safety issue: No ]

Enrollment: 70
Study Start Date: January 2009
Study Completion Date: April 2013
Primary Completion Date: April 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 2
ALS with non-invasive ventilation
Device: non-invasive ventilation
CPAP, BiPAP
No Intervention: 1
ALS without non-invasive ventilation

Detailed Description:

Objectives: Amyotrophic lateral sclerosis is the commonest motor neuron disease with incidence of 0.8 person-years in Chinese. Respiratory muscle function has been proposed to be a strong predictor of quality of life (QoL) and survival in ALS. Some studies suggest that most patients with ALS developed hypoventilation when their vital capacity (FVC) is less than 50% of predicted value. However, the incidence of hypoventilation and factors associated with hypoventilation in ALS patients is not clear. Also, there is still no consensus as to which physiologic marker should be used as a trigger for the initiation of non-invasive ventilation (NIV) in ALS patients. The conflicts of studies come from variable subgroup of ALS, pulmonary function at enrollment, techniques used to diagnose ALS, time to apply NIV, and target endpoint. Therefore, this project aimed to study ALS patients who had relatively preserved respiratory muscle function and no respiratory failure at clinical to achieve three goals: (1) To determine the incidence of hypoventilation in ALS patients (2) To identify the clinical characteristics and risk factors associated with hypoventilation in ALS patients (3) To determine the effect of early intervention with NIV on the prognosis of ALS patients Study design: Randomized, controlled trial Participants: ALS patients whose FVC 40%-80% of predict, Pimax <60mmHg, and daytime PaCO2<50mmHg Protocol: Eligible patients with whole-night polysomnography (PSG) and transcutaneous CO2 (PtcCO2). Enrolled patients were randomized to standard treatment or NIV. The primary endpoint of prognosis was survival. The secondary endpoint was changes of PtcCO2 and PaCO2, unexpected admission or clinic visiting, daytime function and QoL.

Statistic: The baseline demographics of patients with or without hypoventilation were compared to determine the factors associated with hypoventilation in ALS patients. The impact of NIV in ALS patients was determined by comparing the primary and secondary goals between standard treatment and NIV group. A two-sided p value of < 0.05 was considered statistically significant.

Clinical implication: Hypoventilation at ALS patients who had relatively preserved respiratory muscle function and no respiratory failure at clinical sleep was common, early identification through PSG screening and PtcCO2 will allow for the early diagnosis and intervention. Understanding the time of applying NIV and the effect on prognosis in ALS will allow for the early intervention and prediction of outcomes.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • ALS patients who:

    • had FVC 40%-80% of predict
    • Pimax < 60 mmHg
    • daytime PaCO2 < 50 mmHg

Exclusion Criteria:

  • Refuse to participate
  • Require mechanical ventilation
  • Active neurologic event other than ALS
  • Obstructive pulmonary disease
  • Active infection
  • Need sedatives or narcotics within 3 days of sleep study
  • Participating in other study at the same time
  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00958048

Locations
Taiwan
Peilin Lee
Taipei, Taiwan, 100
Sponsors and Collaborators
National Taiwan University Hospital
Taipei Veterans General Hospital, Taiwan
Investigators
Principal Investigator: Peilin Lee, M.D. National Taiwan University Hospital
Principal Investigator: Whey Dong Wu, M.D. Naitonal Taiwan University Hospital
  More Information

No publications provided

Responsible Party: National Taiwan University Hospital
ClinicalTrials.gov Identifier: NCT00958048     History of Changes
Other Study ID Numbers: 200812112R, 2011-04-011LA
Study First Received: February 8, 2009
Last Updated: August 14, 2013
Health Authority: Taiwan: Department of Health

Keywords provided by National Taiwan University Hospital:
Amyotrophic Lateral Sclerosis
hypoventilation

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 31, 2014