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

Primary Purpose

Neuromuscular Disease

Status
Unknown status
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
non invasive mechanical ventilation
Sponsored by
Centre d'Investigation Clinique et Technologique 805
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Neuromuscular Disease focused on measuring Instrumental cough assistance, positive pressure insufflation maneuver, home mechanical ventilation, prospective evaluation

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Home ventilation Unit , Raymond Poincaré hospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

non invasive mechanical ventilation

Arm Description

Patients suffering from neuromuscular disease with NIV indication and cough inefficiency

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

spontaneous vital capacity
spontaneous vital capacity change at 3 months
peak flow
peak cough flow (spontaneous / assisted) change at 3 months
optoelectronic plethysmography
change at 3 months in the distribution in ventilation (assessed by optoelectronic plethysmography), spontaneous/assisted

Full Information

First Posted
November 5, 2014
Last Updated
November 15, 2019
Sponsor
Centre d'Investigation Clinique et Technologique 805
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1. Study Identification

Unique Protocol Identification Number
NCT02288299
Brief Title
Long-Term Effect of LIAM on Respiratory Performance in NIV Patients Suffering From Neuromuscular Disease
Acronym
LIAM
Official Title
Long-Term Effect of LIAM (Lung Insufflation Assist Maneuver) on Respiratory Performance in Non-invasive Ventilated Patients Suffering From Neuromuscular Disease
Study Type
Interventional

2. Study Status

Record Verification Date
February 2019
Overall Recruitment Status
Unknown status
Study Start Date
October 29, 2015 (Actual)
Primary Completion Date
December 31, 2021 (Anticipated)
Study Completion Date
December 31, 2021 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Centre d'Investigation Clinique et Technologique 805

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
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
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neuromuscular Disease
Keywords
Instrumental cough assistance, positive pressure insufflation maneuver, home mechanical ventilation, prospective evaluation

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
non invasive mechanical ventilation
Arm Type
Experimental
Arm Description
Patients suffering from neuromuscular disease with NIV indication and cough inefficiency
Intervention Type
Device
Intervention Name(s)
non invasive mechanical ventilation
Intervention Description
instrumental increase of inspiratory capacity and cough
Primary Outcome Measure Information:
Title
Change in the maximal assisted vital capacity (maximal insufflation capacity) between the begin of the treatment period and the 3-months follow up
Description
Change in the maximal assisted vital capacity (maximal insufflation capacity) between the begin of the treatment period and the 3-months follow up
Time Frame
15 minutes
Secondary Outcome Measure Information:
Title
spontaneous vital capacity
Description
spontaneous vital capacity change at 3 months
Time Frame
15 minutes
Title
peak flow
Description
peak cough flow (spontaneous / assisted) change at 3 months
Time Frame
15 minutes
Title
optoelectronic plethysmography
Description
change at 3 months in the distribution in ventilation (assessed by optoelectronic plethysmography), spontaneous/assisted
Time Frame
1 hour

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
David Orlikowski, MD, PhD
Phone
+33(0)147107777
Email
david.orlikowski@rpc.aphp.fr
First Name & Middle Initial & Last Name or Official Title & Degree
Frederic Lofaso, MD, PhD
Phone
+33(0)147107941
Email
frederic.lofaso@rpc.aphp.fr
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
David Orlikowski, MD, PhD
Organizational Affiliation
Assistance Publique - Hôpitaux de Paris
Official's Role
Principal Investigator
Facility Information:
Facility Name
Home ventilation Unit , Raymond Poincaré hospital
City
Garches
State/Province
Paris Area
ZIP/Postal Code
92380
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
David Orlikowski, MD, PhD
Phone
33147107776
Email
david.orlikowski@rpc.aphp.fr
First Name & Middle Initial & Last Name & Degree
Sandra Pottier, CRA
Phone
33 1 47 10 44 69
Email
sandra.pottier@rpc.aphp.fr
First Name & Middle Initial & Last Name & Degree
Helene Prigent, MD, PhD
First Name & Middle Initial & Last Name & Degree
David Orlikowski, MD, PhD
First Name & Middle Initial & Last Name & Degree
Frederic Lofaso, MD, PhD
First Name & Middle Initial & Last Name & Degree
Adam Ogna, MD, PhD

12. IPD Sharing Statement

Learn more about this trial

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

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