The primary objectives are:*To describe patient characteristics, treatment patterns and the burden of illness over time for individuals in clinical practice with a diagnosis, or suspected diagnosis,2of asthma and/or COPD. Data will be described for…
ID
Source
Brief title
Condition
- Allergic conditions
- Respiratory disorders NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary endpoints are the following, reported for the study population as a
whole, by country, by disease (i.e. asthma, COPD and asthma-COPD overlap when
considered appropriate) and by specified sub-groups:
*Baseline distribution of enrolled patients diagnosed with asthma and/or COPD
as per the physician*s clinical judgement, by disease severity and/or control,
by site type, by physician characteristics, etc.
*Baseline summary statistics (e.g. demographics, physiological, disease
information, productivity/HRQoL, treatments, risk factors, healthcare resources
use, biomarkers, etc.) of patients by disease severity and/or control, by site
type, by physician characteristics, etc.
*Longitudinal (at each follow-up assessment) summary statistics, as suggested
above, of patients by current disease severity and/or control, by site type, by
physician characteristics, etc.
*Identification of phenotypic and endotypic groups, based on clinical
parameters and biomarkers, that are associated with differential outcomes over
time for symptom burden, clinical evolution (including decline in lung
function) and healthcare utilisation. The characteristics of these groups at
baseline and at each follow-up assessment will be described.
*Predictors of phenotypic and endotypic groups, including history of childhood
respiratory symptoms, exposure to tobacco smoke, treatment, etc
*Correlation between biomarkers and phenotypes and endotypes at baseline and at
each follow-up assessment
Secondary outcome
The secondary and exploratory endpoints will include the following, reported
for the study population as a whole, by country, by disease (i.e. asthma, COPD
and asthma-COPD overlap when considered appropriate) and by specified
sub-groups:
*Concordance by severity and/or control between diagnosis according to
guidelines (using the data available at baseline) and according to the initial
clinician diagnosis at recruitment
*Frequency of treatment modification (dosage change, switch, discontinuation)
and reason for modifications between each follow-up assessment
Correlation between biomarkers, disease severity and/or control and different
measures of response to treatment.
*Factors associated with treatment at baseline and patient-reported treatment
satisfaction and preference at baseline and at each follow-up assessment
*Level of symptom control at baseline and at each follow-up assessment
*Frequency of exacerbations according to seasonal variations and conditions,
including RTI, in the year prior to enrolment and during the study
*Summary scores of PROs overall and by specific subgroups (e.g. treatment
patterns, phenotype/endotype, etc.) if considered appropriate, at baseline and
at each follow-up assessment
*Direct and indirect healthcare resource use by resources categories overall
and those related with respiratory disease at baseline and at each follow-up
assessment
*Presence of known risk factors for development of airways disease
*Levels of biomarker parameters, lung function, and risk factors at each
follow-up assessment, and variability in these measures.
*Relationship between disease control, Health-Related Quality of Life (HRQoL),
exacerbations, and healthcare resource use stratified by severity of disease
*Reasons for non-adherence to treatment
Background summary
The two major obstructive lung diseases, asthma and Chronic Obstructive
Pulmonary Disease (COPD), are multifaceted diseases that are associated with
significant impairment and risk of health deterioration, and both currently
have a therapeutic response that is highly variable.
Asthma and COPD have traditionally been viewed as distinct clinical entities.
Recently, however, attention has also been focused on patients with overlapping
features of both asthma and COPD. The concept of the asthma and COPD overlap
focuses attention on the need to accelerate beyond the information available at
present on populations with physician diagnoses of asthma and COPD. The aim
should be to identify phenotypes and endotypes (mechanisms)1to help develop
targeted treatments for patients across the range of obstructive lung diseases
and to generate a new taxonomy of obstructive lung disease. In an attempt to
better characterise and treat asthma and COPD, many studies have been performed
over the last decade to define different phenotypes. A variety of parameters
have been identified for such definitions, including: clinical and functional
characteristics, frequency of exacerbations, age of onset, presence of allergy,
geographical localisation, and different biomarkers. However, many of these
studies have been performed in patients identified by conventional diagnostic
labels, or who satisfy stringent enrolment criteria for clinical trials; the
latter patients may only represent around 4-5% of patients with asthma or COPD
in the general community, and those with asthma-COPD overlap are almost
invariably excluded. In addition, one phenotype may relate to different
endotypes and vice versa so distinct pathogenic profiles need to be more
clearly identified in order to develop personalised treatment strategies
directed towards disease/mechanism-specific features, which can be expected to
improve patients* outcomes.There is a need for new broad and consistent
prospective, observational data on patients with obstructive lung disease,
despite the availability of many current data sources, as well as many
stand-alone respiratory studies and cohorts. This is because very few data
sources span across disease labels, countries or regions and generally do not
collect detailed information on patient characteristics such as patient
reported outcomes (PRO), functional measurements, and healthcare resource use.
Furthermore, there is very limited availability of emerging biomarker data
(especially linked to other data types), which are key to understanding novel
phenotypes and endotypes, disease mechanism, and disease progression, and to
drive scientific discovery in obstructive lung disease.
To overcome these limitations, the NOVELTY study will recruit patients with a
diagnosis or suspected diagnosis of asthma and/or COPD, and collect data
currently lacking to allow for
multinational data collection to fill regional/local gaps and improve
comparability across
regions.
The diversity of data collection approaches will enable capture of rich
clinical data on enrolled patients. The use of advanced statistical analysis
tools will allow characterisation of phenotypes and endotypes associated with
future symptom burden, clinical progression and/or health care utilisation.
Furthermore, it is expected that the data platform created by the study will
continue to be a useful resource even once the objectives of the core study are
met, generating new insight for the broader community of patients, physicians,
payers, regulators, and for the scientific community.
Study objective
The primary objectives are:
*To describe patient characteristics, treatment patterns and the burden of
illness over time for individuals in clinical practice with a diagnosis, or
suspected diagnosis,2of asthma and/or COPD. Data will be described for the
population overall and by pre-specified subgroups, including by country,
demographics, exposures, symptom history, treatment history, concurrent
clinical features, treatment setting, socioeconomic setting and access to
healthcare
*To identify phenotypes and endotypes, based on biomarkers and/or clinical
parameters, that are associated with differential outcomes for symptom burden,
clinical evolution and healthcare resource utilisation, in individuals with a
diagnosis, or suspected diagnosis, of asthma and/or COPD
The secondary objectives are:
*In patients with a diagnosis or suspected diagnosis of asthma and/or COPD, to
compare their current diagnostic labels and physician-assessed disease severity
with existing international criteria and phenotypic groupings
*To describe patient characteristics, symptom burden, quality of life,
exacerbation rates and clinical progression over time, by phenotype/endotype
for: (a) patients with recent-onset chronic respiratory disease, (b) patients
considered by physician assessment or by specified criteria to have mild
disease at enrolment and (c) patients considered by physician assessment or by
specified criteria to have severe disease at enrolment
*To describe the association between specified biomarkers, at enrolment and
over time, and evaluate their stability over time, factors affecting their
variability, and their
relationship with clinical features, among patients with airways disease
Exploratory objectives are:
*To describe healthcare resource use overall and related to respiratory diseases
*To describe PROs, e.g. symptom assessment, disease control, impact on daily
activity
and quality of life
*To assess the occurrence of exacerbations and other conditions, including
upper and lower Respiratory Tract Infections (RTIs), including seasonal
variations, and their relationship with clinical outcomes
*To assess the adequacy of EMR in some countries for obtaining data about
characteristics, clinical progress and treatment of patients with obstructive
airways disease
Exploratory objectives for the Nasal Biospecimen substudy (in the Netherlands
and Sweden) are:
• To investigate nasal mucosal fluid biomarkers in nasal mucosal fluid samples
that will be collected after a Fractional Exhaled Nitric Oxide (FeNO)
measurement
• To perform transcriptomic and epigenetic analyses on RNA and DNA samples,
respectively, taken by nasal epithelium brushings that will be collected after
a FeNO measurement and analysed in conjunction with systemic and nasal
biomarker analyses
Study design
This study is a multi-country3, multicentre, observational, prospective,
longitudinal cohort study which will include patients with a physician
diagnosis, or suspected diagnosis, of asthma and/or COPD. Patients will undergo
clinical assessments and receive standard medical care as determined by the
treating physician. All patients enrolled in the NOVELTY study will be followed
up yearly by their treating physician for a total duration of three years. In
addition, patients are expected to be followed up remotely once every quarter.
A Nasal Biospecimen substudy will be conducted in the Netherlands and Sweden.
This substudy will consist of investigating biomarkers in nasal mucosal fluid
samples collected by nasal absorption; and transcriptomic and epigenetic
analyses on RNA and DNA samples, respectively, taken by nasal epithelial
brushings. These analyses will be interpreted in the context of an FeNO
measurement and blood biomarkers taken at the same visit, as well as in the
context of other available study data.
Study burden and risks
Subjects may choose to provide blood and urine for the study. Furthermore the
subjects will fill-out questionnaires. No further procedures will be performed
with the subjects.
Astraallén Karlebyhus
Södertälje 151 85
SE
Astraallén Karlebyhus
Södertälje 151 85
SE
Listed location countries
Age
Inclusion criteria
· Diagnosis, or suspected diagnosis**, of asthma and/or COPD, according to
clinician's judgement
· Age >=12 years (note: in most countries it will only be feasible to include
patients aged >=18 years)
· Willing and able to sign written, informed consent (or having a responsible,
legally authorised representative acting on patient's behalf)
· Enrolment from an active clinical practice
Exclusion criteria
· Patients who participated in any respiratory interventional trial during the
12 months prior to enrolment or at enrolment
· Patients who, in the opinion of the physician, are unlikely to complete 3
years of
follow-up, e.g. poor literacy, substance abuse, life-threatening co-morbidity
· Patients whose primary respiratory diagnosis (i.e. the condition causing most
of their respiratory symptoms) is not asthma or COPD (however, a co-diagnosis
of another respiratory disease such as bronchiectasis or interstitial lung
disease together with asthma or COPD will be accepted)
In addition, the following are considered criteria for exclusion from the
exploratory genetic research (donation of blood for DNA and RNA analysis)
· Previous allogeneic bone marrow transplant
· Non-leukocyte depleted whole blood transfusion within 120 days of the date of
the genetic sample collection
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL61776.042.17 |