Efficacy and Tolerance of Early Launching of Nocturnal Non Invasive

NCT01225614 | Unknown Phase 3 DMC

• 2 other identifiers: P0812212009-A01023-54 (IDRCB)
• Study Type: interventional
• Target: 77
• Locations: 1 country
• Sites: 1

Brief Summary

This is a multicenter randomized controlled open labeled study testing efficacy and tolerance of early launching of night non invasive ventilation in patients with myotonic dystrophy type 1(DM1). The object of this project is to estimate the effects of the early introduction of non invasive ventilation on the arisen of complication (non expected hospitalization, tracheostomy even death) with regard to a simple respiratory follow-up in patients affected by myotonic dystrophy.

Conditions

Myopathy; Muscular Weakness; Respiratory Insufficiency

Keywords

Home ventilation; Myotonic dystrophy type 1 (DM1)

Outcome Measures

Primary Outcomes (1)

• Rate of patients having a complication (number of non expected hospitalization or death) at 5 years.

  Rate of patients having a complication (number of non expected hospitalization or death) at 5 years.

  5 YEARS

Secondary Outcomes (7)

• Distribution of survival between the randomisation at 5 years

  5 YEARS

• Number of patients having a formal indication of ventilation

  5 YEARS

• Number of tracheostomized patients at 5 years

  5 YEARS

• Number of non expected hospitalizations at 5 years

  5 YEARS

• Observance of the ventilation defined by an average minimal use of 4 am by 12:00 pm .

  5 YEARS

Study Arms (2)

bipap ventilation

EXPERIMENTAL

Device: Bilevel pressure ventilator

Standard care

ACTIVE COMPARATOR

Standard care and ventilation if occurrence of absolute criteria of ventilation (cf infra).

Device: Bilevel pressure ventilator

Interventions

Bilevel pressure ventilator DEVICE

Nocturnal home ventilation

Standard care; bipap ventilation

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Man or woman of age ≥ 18 years
• Preliminary medical examination
• Enlightened and written consent
• Genetically proved Steinert disease
• Presenting at least one of the following 3 criteria
• A hypercapnia: PaCO2 \> 45 mmHg or
• A night-desaturation: SaO2 \< 88 % more than consecutive 5 minutes or
• Apnea syndrome with significant sleep:index of apnea / hypopnea\> a 30 / hour
• And with presence of at least a clinical sign: dyspnoea, orthopnea, headaches, asthenia, diurnal sleepiness, or any other sign suggestive of disturbance of the sleep or of respiratory dysfunction

You may not qualify if:

• Age inferior to 18
• Regime of legal protection
• Pregnancy
• Absolute indication for ventilation: clinical signs (dyspnoea, orthopnea, headaches, asthenia, diurnal sleepiness), AND PaCO2 \> 60 mmHg, AND night-desaturation \< 88 % AND one CV \< 50 % of the theoretical or the PIMAX \< 60 cm H2O
• Acute respiratory failure
• Already ventilated patient
• Patient under oxygen
• Not (beneficiary to a regime of Social Security or legal successor)

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• Association Française contre les Myopathies (AFM), Paris: collaborator

Study Sites (1)

Home ventilation unit and intensive care, centre of neuromuscular disease (Garches Mondor Necker Hendaye), Raymond Poincaré hospital Versailles Saint Quentin University.

Garches, 92380, France

Location

MeSH Terms

Conditions

Muscular Diseases; Muscle Weakness; Respiratory Insufficiency; Myotonic Dystrophy

Condition Hierarchy (Ancestors)

Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Neuromuscular Manifestations; Neurologic Manifestations; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Respiration Disorders; Respiratory Tract Diseases; Muscular Dystrophies; Muscular Disorders, Atrophic; Myotonic Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• DAVID ORLIKOWSKI, MD, PhD

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 20, 2010
• First Posted: October 21, 2010
• Study Start: October 1, 2010
• Primary Completion: July 1, 2018
• Study Completion: December 1, 2018
• Last Updated: November 20, 2017

Record last verified: 2017-11

Locations

FR (1)