* To define the characteristics of patients with severe asthma in terms of clinical features, physiology and biomarker profiles and to compare these with patients with mild-moderate asthma, and healthy controls* To repeat the measurement of clinical…
ID
Source
Brief title
Condition
- Respiratory disorders NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The study will assess and integrate multiple parameters in terms of clinical,
functional, cellular, molecular and patient reported outcomes for developing
phenotype *handprints* of severe asthma. For each parameter a number of
endpoints have been selected which
will be used for the following goals:
* The characterization into phenotypic handprints of patients with severe
asthma and the comparison of these characteristics with those of mild to
moderate asthmatics and healthy controls
* To enable the linking of molecular targets for drug intervention with the
pathophysiology of severe asthma
* Based upon the integration of data collected from severe asthma patients,
classifiers and predictors will be developed. This will form the basis of the
initial phenotype handprint.
Secondary outcome
NA
Background summary
Severe asthmatics are a heterogeneous patient group with frequent exacerbations
and/or progressive airways obstruction despite high levels of therapy.
Development of new treatments for such patients is hampered by heterogeneous
pathogenesis, lack of consensus on diagnostic criteria, difficulty in
translating pre-clinical human and animal models to drug efficacy and absence
of biomarkers that predict therapeutic efficacy.
U-BIOPRED aims to overcome the bottlenecks in drug development for severe
asthma by a focused and stepwise strategy based on an unbiased analysis of
multi-dimensional invasive and non-invasive biomarkers (phenotype handprints).
The use of biomarker profiles comprised of various types of high-dimensional
data from severe asthma cohorts and controls, integrated by an innovative
systems biology approach into distinct phenotype handprints, will enable
significantly better prediction of therapeutic efficacy than single or even
clustered biomarkers of one data type, and will identify novel targets.
Study objective
* To define the characteristics of patients with severe asthma in terms of
clinical features, physiology and biomarker profiles and to compare these with
patients with mild-moderate asthma, and healthy controls
* To repeat the measurement of clinical variables and biomarker profiles during
longitudinal follow up of the severe asthma cohorts, including during severe
exacerbations, and to compare these to baseline measures
* To use the baseline and longitudinal characteristics to determine phenotype
handprints by combining high dimensional datasets using a systems biology
approach
* To develop phenotype handprints which will enable improved clinical
understanding and which will be used to determine clinical progression and
efficacy of interventions
Study design
Multi-centre prospective observational study
Study burden and risks
The burden associated with this study includes several visits, during which an
intake interview, a physical examination, lung function tests will be done.
Bronchoscopy and nasal brushes are optional for subjects. In our own experience
as well as based on literature this is well tolerated by as well mild
asthmatics as patients with moderate to severe asthma.
The patients themselves will not benefit directly from this investigation.
However, the group of asthmatic patients may benefit from this study in the
future. Severe asthmatics are a heterogeneous patient group with frequent
exacerbations and/or progressive airways obstruction despite high levels of
therapy. Development of new treatments for such patients is hampered by
heterogeneous pathogenesis, lack of consensus on diagnostic criteria, of
biomarkers that predict therapeutic efficacy.
U-BIOPRED aims to overcome the bottlenecks in drug development for severe
asthma.
As such, we consider the balance between risks and discomfort for the patients
(low) and the possible benefit for these patient groups in the future
(potentially high) acceptable.
Meibergdreef 9
Amsterdam 1105 AZ
NL
Meibergdreef 9
Amsterdam 1105 AZ
NL
Listed location countries
Age
Inclusion criteria
1. Able to give written informed consent prior to participation in the study, which includes ability
to comply with the requirements and restrictions listed in the consent form. Informed consent
must be obtained prior to undertaking any study procedures.
2. Male or female subject aged 18 years or older at screening.
3. Able to complete the study and all measurements.
4. Able to read, comprehend, and write at a sufficient level to complete study related materials.;The additional in- and exclusion criteria for all cohorts are mentioned on page 28-34 of the protocol.
Exclusion criteria
1. As a result of medical interview, physical examination or screening investigation the physician responsible considers the subject unfit for the study either because of the risk to the patient due to the study or the influence this may have on the study results.
2. The subject has a history of drug or other allergy, which, in the opinion of the responsible physician, contra-indicates their participation.
3. Subject is female who is pregnant or lactating or up to 6 weeks post partum or 6 weeks cessation of breast feeding.
4. The subject has participated within 3 months of the first dose in a study using a new molecular
entity, or the first dose in any other study investigating drugs or having participated within three
months in a study with invasive procedures. Any U-BIOPRED assessments should be deferred
until 3 months after the first dose or invasive procedure. Permission from the Scientific Board must be obtained to enroll or allow the continued participation of a subject enrolled in another
study.
5. Those who, in the opinion of the investigator, have a risk of non-compliance with study procedures.
6. The subject has a recent history of incapacitating psychiatric disorders
7. History or current evidence of an upper or lower respiratory infection or symptoms (including common cold) within 2 weeks of baseline assessments (assessments should be deferred).;The additional in- and exclusion criteria for all cohorts are mentioned on page 28-34 of the protocol.
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 | NL32361.018.10 |