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

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

Effects of Early Intervention With Nocturnal Non-invasive Ventilation in Patients With Amyotrophic Lateral Sclerosis: A Randomized, Controlled Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00958048
• Other study ID #: 200812112R
• Secondary ID: 2011-04-011LA
• Status: Completed
• Phase: N/A
• First received: February 8, 2009
• Last updated: August 14, 2013
• Start date: January 2009
• Est. completion date: April 2013

Study information

• Verified date: August 2013
• Source: National Taiwan University Hospital
• Contact: n/a
• Is FDA regulated: No
• Health authority: Taiwan: Department of Health
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 70
• Est. completion date: April 2013
• Est. primary completion date: April 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Device:
• non-invasive ventilation
CPAP, BiPAP

Locations

Country	Name	City	State
Taiwan	Peilin Lee	Taipei

Sponsors (2)

Lead Sponsor	Collaborator
National Taiwan University Hospital	Taipei Veterans General Hospital, Taiwan

Country where clinical trial is conducted

Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Survival		36 months	No
Secondary	Changes of PtcCO2 and PaCO2, admission or unexpected clinical visiting, and QoL		36 months	No