# Controlling and Preventing Asthma Progression and Severity in Kids with Omalizumab Extension (PARK-EXT)

> **NIH NIH U01** · BOSTON CHILDREN'S HOSPITAL · 2024 · $2,250,000

## Abstract

Project Summary/Abstract
Asthma is the #1 chronic disease in childhood: prevention is an unmet need and pressing priority. We are
conducting the first randomized, double-blind, placebo controlled trial in 200 high risk children 2-3 years of age
to determine whether 2 years of treatment with omalizumab (anti-IgE) will prevent asthma and/or diminish
asthma severity 2 years after treatment is stopped. Our hypothesis revolves around the role that IgE plays in
the development of persistent asthma by catalyzing allergic type 2 driven recurrent wheezing and augmenting
virally-induced exacerbations in susceptible young children. Recent studies have demonstrated the feasibility
and power of using new and novel systems-scale network analysis of transcriptional pathways and epigenetic
signatures to assess airway responses in readily obtained nasal samples. We hypothesize that patients who
respond to anti-IgE may have upregulated expression of genes related to antiviral responses, epithelial cell
structure and barrier integrity. This project will allow us to complete the trial and use state of the art epigenomic
and transcriptomic techniques with integrative approaches to explore this hypothesis and examine the role of
specific alterations in Treg cells and antigen-specific T cells in transducing clinical responses seen in the trial.
Our first aim is to complete the trial to assess whether interfering with environmental allergen IgE-mediated
immunological responses can prevent or moderate a progression to asthma in susceptible children. Our
second aim is to examine the relationship between anti-viral and epithelial integrity pathways and the response
to anti-IgE through DNA methylation and transcriptomic signatures and associated effects on Type 2
inflammation, wheezing episodes, asthma, and asthma severity. We will extend our findings to include single-
cell transcriptional profiling in a subset to more fully inform the cellular sources of bulk transcriptome responses
and provide important molecular details about individual cell heterogeneity and rare cell populations that relate
to clinical responsiveness and clinical outcomes. We will integrate multi-omic and clinical data and perform
analyses to define the combined transcriptomic and epigenetic changes underlying response to anti-IgE
treatment and the persistence or resolution of benefit (including asthma outcome) following cessation of
therapy. These signatures will provide comprehensive mechanistic insights on clinical treatment effects of
responders vs. non-responders, and specific DNA methylation and gene expression molecular pathways that
impact study outcomes and elucidate the disease modifying effects of anti-IgE. Differences in these profiles
could provide potential biomarkers predictive of clinical and immunologic response to anti-IgE. This study is
potentially paradigm shifting – regardless of the outcomes of the trial, we will gain considerable knowledge on
the pathophysiology of the disease a...

## Key facts

- **NIH application ID:** 10764438
- **Project number:** 1U01AI179563-01
- **Recipient organization:** BOSTON CHILDREN'S HOSPITAL
- **Principal Investigator:** WANDA PHIPATANAKUL
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $2,250,000
- **Award type:** 1
- **Project period:** 2024-07-08 → 2029-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10764438

## Citation

> US National Institutes of Health, RePORTER application 10764438, Controlling and Preventing Asthma Progression and Severity in Kids with Omalizumab Extension (PARK-EXT) (1U01AI179563-01). Retrieved via AI Analytics 2026-06-15 from https://api.ai-analytics.org/grant/nih/10764438. Licensed CC0.

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