Asthma
Conditions
Keywords
asthma, phenotyping, transcriptomics, network analysis, genomics, biologics
Brief summary
A retrospective and prospective observational study of asthma designed to determine real-world and molecular description of asthma according to treatment and type 2 biology
Detailed description
REGAIN is a retrospective and prospective asthma cohort study designed to determine real-world molecular description of asthma according to treatment and type 2 biology. The researchers will enroll 5 pre-specified groups of participants with asthma of specific interest for mechanistic analyses according to clinically determined type 2 inflammatory endotype and response to therapy with inhaled steroids and other inhaled controllers with or without asthma biologics. This study is conducted at asthma specialty programs at Mount Sinai National Jewish Health Respiratory Institute in New York and National Jewish Health, Denver, Colorado, USA. Participants with asthma meeting specific inclusion criteria were identified and enrolled by screening electronic medical records from pulmonary and allergy specialty programs. Targeted populations included 5 categories of asthma patients as outlined in Table 1 including the following: (1) patients with type 2 asthma with symptom control at time of enrollment (ACT \> 20) on standard Global Initiative For Asthma (GINA) step therapy (n=200) (2) asthma managed on stable biological therapy (n=200), (3) type 2 asthma with prior treatment with at least 2 biological therapies, (n=60), (4) likely type 2 low asthma patients (n=200), (5) asthma patients scheduled to be started de novo on biological therapies (n=120), and (6) healthy participants (n=400). Healthy participants were recruited in New York and Denver by community advertising. An optional study component employing a mobile phone application, use of digital inhaler monitors, and home digital peak flow will contribute data regarding medication use and lung function in the community and collect environmental data impacting asthma using geolocation data.
Interventions
None listed
Sponsors
Icahn School of Medicine at Mount Sinai
Sanofi
GeneDx
National Jewish Health
Study design
Observational model
CASE_CONTROL
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
Yes
Inclusion criteria
for Asthma Participants: * Age \>18 * Clinical diagnosis of asthma verified by a specialist physician (allergist or pulmonologist) and one or more of the following: * Historical variability of airflow limitation via classic reversibility criteria (\> 12%, 200 ml within past 10 years). * Historical methacholine challenge with PC20 \< 8 mg/ml (within past 10 years). * FEV1 variability, between two clinic visits, of 20% within the past 5 years either with improvement according to appropriate therapy or decrease in lung function upon withdrawal or decrease of therapy. * Elevated FeNO \> 50 ppb at least once historically (within past 10 years). * \< 10 pack per year personal tobacco use and not a current smoker of cigarettes or other inhalational tobacco (e-cigarette, marijuana or other inhalational drug use). * At least partial response of presenting symptoms to GINA Step 1-4 asthma treatment (ICS or ICS/LABA as determined by study physician investigators)
Exclusion criteria
for Asthma Participants: * \>10 pack per year tobacco, current smoker of cigarettes, e-cigarettes or other inhalational drug use. * Clinically significant lung disease based on imaging or clinical history other than asthma as determined by an allergist or pulmonologist * Currently pregnant or breastfeeding * Anemia with hemoglobin level ˂9 mg/dl at the time of study enrollment Inclusion Criteria for Healthy Controls: * Age \> 18 * No current smoking (quit \> 6 months previously) confirmed by serum cotinine level \< 1 ng/ml * \< 10 pack year tobacco history * No current respiratory symptoms (cough wheeze, dyspnea) * No current lung disease or other significant medical comorbidity (see
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Asthma disease subtypes | 18 months | Description of asthma disease subtypes according to type 2 and non-type 2 biology and control or lack of control along a range of asthma treatments. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of responders versus non-responders | 6 and 18 months | Control on GINA Step 1-5 therapy will be defined by an ACT \>20. Control on biological therapies will be defined by ACT \> 20, or if ACT 16-19, achieving "partly controlled" status in according to GINA, or achieving a 50% reduction in oral corticosteroid (OCS) dose if patient is OCS dependent. |
| Symptom status | 6 and 18 months | The proportion of participants achieving or maintaining symptom control, exacerbation control, both of these, or those achieving clinical remission at 6 and 18 months among asthma subgroups |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORLinda Rogers, MD
Icahn School of Medicine at Mount Sinai
PRINCIPAL_INVESTIGATORKe Hao, PhD
Icahn School of Medicine at Mount Sinai
PRINCIPAL_INVESTIGATOREileen Wang, MD
National Jewish Health
Outcome results
None listed