Real World and Genomic Data Based Asthma Insight Through Network Analysis

NCT06623435 | Active Not Recruiting

• 1 other identifier: GCO 19-0358
• Study Type: observational
• Target: 1,073
• Locations: 1 country
• Sites: 5

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

Conditions

Asthma

Keywords

asthma; phenotyping; transcriptomics; network analysis; genomics; biologics

Outcome Measures

Primary Outcomes (1)

• Asthma disease subtypes

  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.

  18 months

Secondary Outcomes (2)

• Number of responders versus non-responders

  6 and 18 months

• Symptom status

  6 and 18 months

Study Arms (6)

Type 2 Step Therapy Asthma

Participants in this group must meet criteria for asthma diagnosis and type 2 asthma status (1) Exhaled nitric oxide (FeNO) \> 50 ppb at any clinical care time point (2) at least one blood eosinophil count (BEC) \> 300/uL at any clinical care time point (3) eosinophilia by induced sputum or bronchoscopy (\> 3%) if available historically, performed as part of clinical care) and be on inhaled step therapy according to the Global Initiative for Asthma 2018 Step Therapy 1-5 treatment. They can not be on chronic oral corticosteroids or biologic therapy and must have an Asthma Control test (ACT) of 20 or greater.

Likely Type 2 Low Step Therapy Asthma

Participants with likely type 2 low asthma (exhaled nitric oxide-FeNO \< 25 ppb AND blood eosinophil count (BEC) \< 200/uL AND no eosinophilia by induced sputum or bronchoscopy if available historically or as obtained during clinical care. On inhaled step therapy according to the Global Initiative for Asthma 2018 Step Therapy 1-5 treatment. They cannot be on chronic oral corticosteroids or biologic therapy and can have any value for Asthma Control test (ACT)

Stable Biologics Asthma

Participants included in the stable biological cohort must meet criteria for asthma diagnosis. The patients must also be on biological therapy for at least 4 months at enrollment. These participants needed to have an ACT of 20 or greater at time of enrollment, or if ACT 16-19 but they had GINA symptom control level 1-2, then they are considered partly controlled and were included in this group.

De Novo Biologics Asthma

Either 2 type 2 or type 2 low characteristics Asthma participants with uncontrolled asthma on inhaled therapy (Global Initiative for Asthma 2018) Any asthma control test score (ACT) Starting a clinically prescribed biologic therapy for asthma Not on another biologic therapy for at least 4 months

Failed Multiple Biologics Asthma

Either 2 type 2 or type 2 low characteristics. Participants included in this group must report prior biological treatment and having stopped treatment with at least 2 prior asthma biologics. They could be on or off biologic therapy at enrollment and have any level of asthma control by asthma control test and GINA at the time of enrollment.

Healthy participants

Healthy individuals with no respiratory symptoms

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Specific groups of asthma patients as outlined and healthy volunteers

You may qualify if:

• 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)

You may not qualify if:

• \>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
• 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)
• Age \< 18
• Diagnosis of asthma, COPD, emphysema, bronchiectasis or other significant lung disease
• Treatment or a history of malignancy currently or within the past 5 years, except completely treated in situ carcinoma of the cervix, completely treated and resolved non-metastatic squamous or basal cell carcinoma of the skin
• Treatment of inflammatory disease or autoimmune disease such as lupus, rheumatoid arthritis, inflammatory bowel disease, multiple sclerosis or others
• Treatment with immune modulating medications for a health condition including any of the following (systemic glucocorticoids, sirolimus, tacrolimus, anti-TNF abatacept, azathioprine, methotrexate, cyclosporine, cyclophosphamide, immunoglobulin, mycophenolate, rituximab, plaquenil or others)
• Treatment with anti-IL5, anti-IgE and anti-IL4 receptor alpha for non-asthma indications
• Vaccination within 4 weeks

Sponsors & Collaborators

• Icahn School of Medicine at Mount Sinai: lead
• Sanofi: collaborator
• GeneDx: collaborator
• National Jewish Health: collaborator

Study Sites (5)

National Jewish Health

Denver, Colorado, 80206, United States

Location

Mount Sinai Beth Israel

New York, New York, 10003, United States

Location

Mount Sinai West

New York, New York, 10019, United States

Location

Mount Sinai Morningside

New York, New York, 10027, United States

Location

Mount Sinai Hospital

New York, New York, 10029, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Nasal brushings for RNA transcriptomics Peripheral blood for genomics, proteomics and storage of peripheral blood mononuclear cells

MeSH Terms

Conditions

Asthma

Condition Hierarchy (Ancestors)

Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Respiratory Hypersensitivity; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases

Study Officials

• Linda Rogers, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

• Ke Hao, PhD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

• Eileen Wang, MD

  National Jewish Health

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: September 30, 2024
• First Posted: October 2, 2024
• Study Start: November 7, 2019
• Primary Completion: December 31, 2025
• Study Completion: December 31, 2025
• Last Updated: September 4, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: Specify Other Time FrameAfter last participant completes the study and publication.
• Access Criteria: Investigators whose proposed use of the data has been approved by an independent review committee ('learned intermediary') identified for this purpose. To achieve aims in the approved proposal. Specify Other Mechanism Please email requests for access to linda.rogers@mssm.edu or Emanuele.deRinaldis@sanofi.com

Locations

US (5)