Long-Term Effect of LIAM on Respiratory Performance in NI... | NCT02288299

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

NCT02288299

Sponsor

Centre d'Investigation Clinique et Technologique 805

Status

Unknown status

Last Update Posted

Nov 18, 2019Actual

Enrollment

30Estimated

Phase

Not Applicable

Conditions

Neuromuscular Disease

Interventions

non invasive mechanical ventilation

Countries

France

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Feb 2019

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Oct 29, 2015Actual

Primary Completion Date

Dec 31, 2021Estimated

Completion Date

Dec 31, 2021Estimated

First Submitted Date

Nov 5, 2014

First Submission Date that Met QC Criteria

Nov 7, 2014

First Posted Date

Nov 11, 2014Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Nov 15, 2019

Last Update Posted Date

Nov 18, 2019Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

30Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
non invasive mechanical ventilation	Experimental	Patients suffering from neuromuscular disease with NIV indication and cough inefficiency	Device: non invasive mechanical ventilation

Interventions

Name	Type	Description	Arm Group Labels	Other Names
non invasive mechanical ventilation	Device	instrumental increase of inspiratory capacity and cough	non invasive mechanical ventilation

Outcomes Module

Primary Outcomes

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 Outcomes

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

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
David Orlikowski, MD, PhD	Contact	+33(0)147107777		david.orlikowski@rpc.aphp.fr
Frederic Lofaso, MD, PhD	Contact	+33(0)147107941		frederic.lofaso@rpc.aphp.fr

Overall Officials

Name	Affiliation	Role
David Orlikowski, MD, PhD	Assistance Publique - Hôpitaux de Paris	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Home ventilation Unit , Raymond Poincaré hospital	Recruiting	Garches	Paris Area	92380	France

ID	Term
D009468	Neuromuscular Diseases

ID	Term
D009422	Nervous System Diseases

Intervention Browse Module

MeSH Terms

ID	Term
D063087	Noninvasive Ventilation

Ancestor Terms

ID	Term
D012121	Respiration, Artificial
D058109	Airway Management
D013812	Therapeutics
D012138	Respiratory Therapy

Browse Leaves

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Browse Branches

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