Clinical trial NCT02315339: European Home Mechanical Ventilation Registry
Official title: European Home Mechanical Ventilation Registry
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Pulmonary Disease, Chronic Obstructive
Amyotrophic Lateral Sclerosis
Spinal Cord Injury
Muscular Dystrophies
Obesity Hypoventilation Syndrome
Kyphoscoliosis
Congenital Central Hypoventilation Syndrome
Duchenne Muscular Dystrophy
Myopathies
Myotonic Dystrophy
Lead sponsor: ResMed
Sponsor class: INDUSTRY
Collaborators: name: Clinical Trial Center North Hamburg Germany
name: CRI-The Clinical Research Institute GmbH
First posted: 2014-12-11
Enrollment: 37
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The registry will target all adult individuals who have an indication for HMV (including those with amyotrophic lateral sclerosis \[ALS\], central hypoventilation syndrome \[CHS\], chronic obstructive pulmonary disease \[COPD\], kyphoscoliosis, obesity hypoventilation syndrome \[OHS\], spinal cord injury \[SCI\], Duchenne muscular dystrophy \[DMD\], muscular dystrophies \[MDs\] other than DMD, myopathies and myotonic dystrophy \[Steinert's muscular dystrophy; SMD\]). All newly-diagnosed patients with chronic respiratory diseases who are eligible for HMV treatment as part of routine care can be included in the registry. Patients who satisfy all the inclusion criteria will be consecutively enrolled into the registry.

Brief summary: The European Home Mechanical Ventilation Registry (EHMVR) will enable a thorough evaluation of HMV by documenting the characteristics of HMV patients and their treatment. This will facilitate a prospective, observational study to identify the primary indications for HMV, describe patterns of HMV use in European countries, and characterize changes in the initiation and utilization of HMV over time. The registry will target all adult individuals who have an indication for HMV. In the EHMVR, patient data from routine clinical care will be documented using an electronic case report form (eCRF). The eCRF will record: patient demographic data; diagnostic information (including primary diagnosis, 6-minute walk time, the presence of depression, and quality of life); blood gases; ventilation treatment (including type of ventilator, modes and settings, interfaces used); follow-up data (including failure rates, side effects, technical issues). An initial Pilot Phase will be launched with the aim to enrol at least 200 patients over a 6-month period to determine the feasibility of the registry. Steering committee members and their institutions will be the main participants in the Pilot Phase. After completion of the Pilot Phase, the registry will be expanded across Europe with the goal of enrolling approximately 10,000 patients over 5 years.

Detailed description: The data (baseline and follow-up) should be collected as part of routine clinical care. In clinical practice, after HMV initiation, the first control examination (follow-up number one) with nocturnal diagnostics should take place within the first 4-8 weeks. Annual follow-up is required, and it is recommended that subsequent control visits should be performed 1-2 times a year. Each patient will be included for 5 years (total duration of the registry).

Eligibility criteria: Inclusion Criteria:

* Patients aged ≥18 years with an indication to receive HMV as part of routine clinical care
* Patient is able to fully understand the study information and is willing to give informed consent
* Patient, or the patient's legal guardian, signing the consent form

Exclusion Criteria:

- No exclusion criteria have been defined because only data from routine clinical care are needed, plus a separate healthcare questionnaire

Primary outcomes:
measure: Determine the mortality rate and the number of hospital readmissions
timeFrame: every year (2014 to 2019), up to 5 years
Secondary outcomes:
measure: Determine the effects of home mechanical ventilation on health-related quality of life using the EQ-5D and the Severe Respiratory Insufficiency questionnaires quality of life
timeFrame: every year (2014 to 2019), up to 5 years
Other outcomes:
Locations:
city: Cologne
country: Germany
facility: Lungen Klinik - Chefarzt Abt. Pneumologie Kliniken der StadtKölng GmbH
Study officials:
affiliation: Laboratoire d'EFCR, RDC Haut Chartreuse, CHU Michallon 38043,Grenoble, France
name: Jean-Louis Pepin, Pr
role: PRINCIPAL_INVESTIGATOR
affiliation: Pneumologia e Terapia Intensiva Respiratoria Ospedale Sant' Orsola Malpighi, Italy
name: Stefano Nava, Pr
role: PRINCIPAL_INVESTIGATOR
affiliation: NIHR Respiratory Biomedical Research Unit, Royal Brompton and Harefield NHS Foundation Trust, United Kingdom
name: Anita Simonds, Pr
role: PRINCIPAL_INVESTIGATOR
affiliation: Lungen Klinik - Chefarzt Abt. Pneumologie Kliniken der StadtKölng GmbH, Ostmerheimer Strasse 200, Germany
name: Wolfram Windisch, Pr
role: PRINCIPAL_INVESTIGATOR
References: