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.
Bronchiectasis is an increasingly prevalent chronic pulmonary disorder that is often caused by medical conditions such as primary ciliary dyskinesia (PCD), primary immunodeficiencies (PID), alpha-1 anti-trypsin disease (AATD) and cystic fibrosis (CF). Research on bronchiectasis is limited, and large knowledge gaps remain regarding the epidemiology, pathophysiology, and treatment options. The proposed conference, the first of its kind, will provide an opportunity for experts across the globe to convene for the purposes of knowledge sharing and strategic planning for collaborative multicenter trials, with planned post-conference publications and videos for disseminating information to the scientific community, patients, and families.
