Long-Term Effect of LIAM on Respiratory Performance in NIV Patients Suffering From Neuromuscular Disease (LIAM)

Long-Term Effect of LIAM (Lung Insufflation Assist Maneuver) on Respiratory Performance in Non-invasive Ventilated Patients Suffering From Neuromuscular Disease

The long-term effect of LIAM (Lung Insufflation Assist Maneuver) on respiratory performance in home non-invasively ventilated (NIV) patients suffering from neuromuscular disease will be assessed in a prospective, randomized, cross over, open label study

Study Overview

• Status: Unknown
• Conditions: Neuromuscular Disease
• Intervention / Treatment: Device: non invasive mechanical ventilation

Detailed Description

In advanced neuromuscular disorders, respiratory complications represent the main cause of morbidity and mortality. Beside chronic respiratory insufficiency, necessitating a ventilatory support, mostly performed by non-invasive ventilation (NIV), cough is impaired due to the muscle weakness, and respiratory physiotherapy becomes an essential part of the management.

Various techniques have been proposed to improve lung recruitment and cough in neuromuscular patients. The assisted techniques based on a positive pressure insufflation maneuver have shown an improvement in physiological variables on the short term, but there is to date no good-quality prospective study allowing to evaluate the long term efficacy of mechanical cough assistance devices in neuromuscular patients.

We designed a randomized, cross over, open label study to assess the long-term effect of LIAM (Lung Insufflation Assist Maneuver) on respiratory performance in NIV patients suffering from neuromuscular disease.

• Study Type: Interventional
• Enrollment (Anticipated): 30
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: David Orlikowski, MD, PhD; Phone Number: +33(0)147107777; Email: david.orlikowski@rpc.aphp.fr
• Study Contact Backup: Name: Frederic Lofaso, MD, PhD; Phone Number: +33(0)147107941; Email: frederic.lofaso@rpc.aphp.fr

Study Locations

• France
  • Paris Area
    • Garches, Paris Area, France, 92380
      • Recruiting
      • Home ventilation Unit , Raymond Poincaré hospital
      • Contact: David Orlikowski, MD, PhD; Phone Number: 33147107776; Email: david.orlikowski@rpc.aphp.fr
      • Contact: Sandra Pottier, CRA; Phone Number: 33 1 47 10 44 69; Email: sandra.pottier@rpc.aphp.fr
      • Sub-Investigator: Hélène Prigent, MD, PhD
      • Principal Investigator: David Orlikowski, MD, PhD
      • Sub-Investigator: Frederic Lofaso, MD, PhD
      • Sub-Investigator: Adam Ogna, MD, PhD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• o neuromuscular disease ( progressive muscular dystrophy, for example; Duchenne muscular dystrophy, spinal muscular atrophy)

  • age ≥ 18 years
  • indication to NIV (ongoing ventilation or new patients)
  • vital capacity ≤ 50% pred
  • peak cough flow < 270 l/min
  • "LIAM Responder" = increased insufflation capacity (≥ + 50%) and Peak Cough Flow (PCF ≥+ 50%) with LIAM

Exclusion Criteria:

• o acute respiratory failure (respiratory acidosis)

  • home treatment by instrumental cough assistance in the preceding 12 months
  • ongoing medical treatment of the neuromuscular disease (for ex: corticosteroids in Duchenne disease, enzyme therapy in Pompe disease)
  • previous pneumothorax
  • plan of legal protection
  • pregnant or breastfeeding women
  • failure to cooperate
  • no affiliation to a social security scheme

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: non invasive mechanical ventilation; Patients suffering from neuromuscular disease with NIV indication and cough inefficiency	Device: non invasive mechanical ventilation; instrumental increase of inspiratory capacity and cough

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in the maximal assisted vital capacity (maximal insufflation capacity) between the begin of the treatment period and the 3-months follow up	Change in the maximal assisted vital capacity (maximal insufflation capacity) between the begin of the treatment period and the 3-months follow up	15 minutes

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
spontaneous vital capacity	spontaneous vital capacity change at 3 months	15 minutes
peak flow	peak cough flow (spontaneous / assisted) change at 3 months	15 minutes
optoelectronic plethysmography	change at 3 months in the distribution in ventilation (assessed by optoelectronic plethysmography), spontaneous/assisted	1 hour

Collaborators and Investigators

• Sponsor: Centre d'Investigation Clinique et Technologique 805
• Investigators: Principal Investigator: DAVID ORLIKOWSKI, MD, PhD, Assistance Publique - Hôpitaux de Paris

Study record dates

Study Major Dates

• Study Start (Actual): October 29, 2015
• Primary Completion (Anticipated): December 31, 2021
• Study Completion (Anticipated): December 31, 2021

Study Registration Dates

• First Submitted: November 5, 2014
• First Submitted That Met QC Criteria: November 7, 2014
• First Posted (Estimate): November 11, 2014

Study Record Updates

• Last Update Posted (Actual): November 18, 2019
• Last Update Submitted That Met QC Criteria: November 15, 2019
• Last Verified: February 1, 2019

More Information

Terms related to this study

• Keywords: home mechanical ventilation; Instrumental cough assistance; positive pressure insufflation maneuver; prospective evaluation
• Additional Relevant MeSH Terms: Nervous System Diseases; Neuromuscular Diseases
• Other Study ID Numbers: 2014-A01005-42