# Rare Genetic Causes of Bronchiectasis: Paving the Way for Interventional Trials

> **NIH NIH R13** · SEATTLE CHILDREN'S HOSPITAL · 2021 · $19,959

## Abstract

Abstract
The objective of the proposed conference is to bring together clinical and translational
investigators in rare genetic causes of bronchiectasis, including primary ciliary dyskinesia (PCD),
primary immunodeficiencies (PID), alpha-1 anti-trypsin disease (AATD) and cystic fibrosis (CF),
to discuss preparing for collaborative multicenter clinical trials. In this day and a half-long
conference, presenters will address critical knowledge gaps in disease pathogenesis, clinical trial
preparedness and overcoming barriers to successful trials in these rare disease populations.
Speakers will provide state of the art updates on the diagnosis and major sources of morbidity in
these conditions, as well as commonalities and differences in bronchiectasis pathogenesis, with
an emphasis on potential therapeutic targets. Lessons learned from previous clinical trials,
particularly in CF, will be presented, in order to capitalize on previous experience as we plan
future clinical trials. The strengths and weaknesses of potential clinical trial endpoints will be
discussed. Innovative study designs for rare diseases and partnering with stakeholders will be
highlighted. Junior investigators will be invited to present abstracts at a poster session and will
participate in special sessions regarding career development and funding opportunities. The
conference will promote interaction and discussion, with question and answer sessions after each
presentation and panel discussions. The expected conference output will be a series of peer-
reviewed manuscripts summarizing the meeting sessions, with an overarching theme of paving
the way for successful clinical trials in these rare disease populations. Video summaries of
relevant contents directed towards patients and families will also be produced and disseminated
following the conference.

## Key facts

- **NIH application ID:** 10144683
- **Project number:** 1R13HL156477-01
- **Recipient organization:** SEATTLE CHILDREN'S HOSPITAL
- **Principal Investigator:** BreAnna Kinghorn
- **Activity code:** R13 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $19,959
- **Award type:** 1
- **Project period:** 2021-02-15 → 2022-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10144683

## Citation

> US National Institutes of Health, RePORTER application 10144683, Rare Genetic Causes of Bronchiectasis: Paving the Way for Interventional Trials (1R13HL156477-01). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10144683. Licensed CC0.

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