Clinical trial NCT04240925: Tolerability, Safety and Efficacy of Sigh Breaths During NIMV in Motor Neuron Disease
Official title: Tolerability, Safety and Efficacy of Sigh During Non-invasive Mechanical Ventilation in Patients With Motor Neuron Disease- A Pilot Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Motor Neuron Disease
Respiratory Failure
Amyotrophic Lateral Sclerosis
Lead sponsor: Ospedale San Raffaele
Sponsor class: OTHER
First posted: 2020-01-27
Enrollment: 44
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Changes in nocturnal oxygen desaturation index (ODI) assessed by transcutaneous nocturnal oximetric registration
timeFrame: Evaluation will be performed before NIMV initiation and two months after
Secondary outcomes:
measure: Changes in the arterial blood PCo2 concentration assessed by arterial blood gas test
timeFrame: Evaluation will be performed before NIMV initiation and two months after
measure: Changes in the arterial blood PO2 concentration assessed by arterial blood gas test
timeFrame: Evaluation will be performed before NIMV initiation and two months after
measure: Changes in the arterial blood Ph assessed by arterial blood gas test
timeFrame: Evaluation will be performed before NIMV initiation and two months after
measure: Changes in the forced vital capacity (FVC) assessed by spirometry test
timeFrame: Evaluation will be performed before NIMV initiation and two months after
measure: 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)
timeFrame: Evaluation will be performed before NIMV initiation and two months after
measure: 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.
timeFrame: Evaluation will be performed before NIMV initiation and two months after
measure: Changes in sleep quality assessed by Numeric Rating Scale (NRS). Score between 0 to 10, where higher scores denote a worse sleep quality.
timeFrame: Evaluation will be performed before NIMV initiation and two months after
measure: Changes in nocturnal dyspnoea assessed by Numeric Rating Scale (NRS). Score between 0 to 10, where higher scores denote a worse nocturnal dyspnoea.
timeFrame: Evaluation will be performed before NIMV initiation and two months after
Other outcomes:
Locations:
city: Milano
country: Italy
facility: Ospedale San Raffaele
Study officials:
affiliation: Ospedale San Raffaele
name: nilo riva, MD,PhD
role: PRINCIPAL_INVESTIGATOR
References: