Tolerability, Safety and Efficacy of Sigh Breaths During NIMV in Motor Neuron Disease

Tolerability, Safety and Efficacy of Sigh During Non-invasive Mechanical Ventilation in Patients With Motor Neuron Disease- A Pilot Study

Non-invasive mechanical ventilation (NIMV) is the recommended standard of care as initial therapy for patients with motor neuron disease (MND) with deterioration of the respiratory function.

SIGH_01 study is aimed at investigating the tolerability, safety profile and efficacy of sigh breaths during non-invasive mechanical ventilation in patients with MND in comparison to the standard ventilation support protocol.

Study Overview

• Status: Active, not recruiting
• Conditions: Respiratory Failure; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Device: NIMV with sigh breaths; Device: Standard NIMV

• Study Type: Interventional
• Enrollment (Anticipated): 44
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Italy
  • Milano, Italy, 20132
    • Ospedale San Raffaele

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: ADULT; OLDER_ADULT; CHILD
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Willing and able to give informed consent
• MND diagnosis according to El-Escorial criteria
• Non-invasive ventilation indications in accordance with the international guidelines

Exclusion Criteria:

• Inability to adhere to study visit schedule or lack of reliable caretaker
• Presence of dementia
• History of arrhythmia, heart failure or pneumothorax

Study Plan

How is the study designed?

Design Details

• Primary Purpose: SUPPORTIVE_CARE
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: TRIPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Standard NIMV protocol with sigh breaths	Device: NIMV with sigh breaths; Sigh breaths: Maximal Inspiratory pressure (IPAPmax) increased of at least 25% ( ≥ 125%) with a frequency of at least 1/200 breaths. The sigh breaths will be delivered as part of the standard NIMV protocol
ACTIVE_COMPARATOR: Standard NIMV protocol without sigh breaths	Device: Standard NIMV; Will be treated by standard NIMV with no sigh

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Changes in nocturnal oxygen desaturation index (ODI) assessed by transcutaneous nocturnal oximetric registration	Evaluation will be performed before NIMV initiation and two months after

Secondary Outcome Measures

Outcome Measure	Time Frame
Changes in the arterial blood PCo2 concentration assessed by arterial blood gas test	Evaluation will be performed before NIMV initiation and two months after
Changes in the arterial blood PO2 concentration assessed by arterial blood gas test	Evaluation will be performed before NIMV initiation and two months after
Changes in the arterial blood Ph assessed by arterial blood gas test	Evaluation will be performed before NIMV initiation and two months after
Changes in the forced vital capacity (FVC) assessed by spirometry test	Evaluation will be performed before NIMV initiation and two months after
Changes in self-perceived quality of life(QoL) assessed by 5-item Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-5). Score between 0 and 100 pts (higher score denotes worse QoL)	Evaluation will be performed before NIMV initiation and two months after
Changes in sleep quality assessed by Pittsburg Sleep Quality Index (PSQI) questionnaire. Score between 0 to 21, where higher scores denote a worse sleep quality.	Evaluation will be performed before NIMV initiation and two months after
Changes in sleep quality assessed by Numeric Rating Scale (NRS). Score between 0 to 10, where higher scores denote a worse sleep quality.	Evaluation will be performed before NIMV initiation and two months after
Changes in nocturnal dyspnoea assessed by Numeric Rating Scale (NRS). Score between 0 to 10, where higher scores denote a worse nocturnal dyspnoea.	Evaluation will be performed before NIMV initiation and two months after

Collaborators and Investigators

• Sponsor: Ospedale San Raffaele
• Investigators: Principal Investigator: nilo riva, MD,PhD, Ospedale San Raffaele

Study record dates

Study Major Dates

• Study Start (ACTUAL): May 25, 2018
• Primary Completion (ANTICIPATED): April 30, 2022
• Study Completion (ANTICIPATED): December 31, 2022

Study Registration Dates

• First Submitted: January 16, 2020
• First Submitted That Met QC Criteria: January 23, 2020
• First Posted (ACTUAL): January 27, 2020

Study Record Updates

• Last Update Posted (ACTUAL): March 16, 2022
• Last Update Submitted That Met QC Criteria: March 15, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Respiratory Tract Diseases; Respiration Disorders; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Respiratory Insufficiency; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: SIGH_01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No