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

September 23, 2021 to September 24, 2021

This conference will 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 participate in special sessions regarding career development and funding opportunities. Patients and families will have the opportunity to participate in the conference sessions, as well as “Meet the Expert” sessions.

Target Audience

The target audience for this conference is clinical and translational investigators (MD/DO, PhD) 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. We will also have a Career Enhancement Curriculum for trainees/junior faculty and a Family Curriculum as part of the conference.

International Participants

International participants are welcome! Please note the mobile number feature is not enabled for international guests. International guests, please complete the registration process by visiting the "Register" tab.

Learning Objectives

  1. Discuss and summarize critical knowledge gaps and key steps for clinical trial readiness in these rare disease populations
  2. Create collaborative working groups to propose and develop future clinical trials 
  3. Excite and promote the success of junior investigators in rare disease clinical and translational research
  4. Facilitate patient and family involvement in both research sessions and family focused “Meet the Expert” sessions

Additional Information

Course Contact: 
Matthew Stern, MS | CME Manager | Children's Hospital Colorado | matthew.stern@childrenscolorado.org
Course summary
Available credit: 
  • 8.75 AMA PRA Category 1 Credit™
  • 8.75 Attendance
Course opens: 
06/15/2021
Course expires: 
10/08/2021
Event starts: 
09/23/2021 - 7:00am
Event ends: 
09/24/2021 - 4:30pm
Cost:
$20.00
Rating: 
0

Day 1 - September 23, 2021:

8:00-8:10am     Welcome and Introduction - BreAnna Kinghorn, Margaret Rosenfeld (co-chairs)

8:10-10:15am   Rare Genetic Causes of Bronchiectasis: Clinical Spectrum
     
Session Moderators: Adam Shapiro, MD, Steve Lommatzsch, MD

  • 8:10-8:30: Overview of Rare Genetic Causes of Bronchiectasis : Steve Lommatzsch, MD
    Live Q&A 8:30-8:35
  • 8:35-8:55: Cystic Fibrosis—the Model: Deepika Polineni, MD
    Live Q&A 8:55-9:00
  • 9:00-9:20: Primary Ciliary Dyskinesia: Stephanie Davis, MD
    Live Q&A 9:20-9:25
  • 9:25-9:45: Primary Immune Deficiencies: Megan Cooper, MD
    Live Q&A 9:45-9:50
  • 9:50-10:10: Alpha-1 Antitrypsin Deficiency: Robert Sandhaus, MD, PhD
    Live Q&A 10:10-10:15

10:15-10:30am   Break

10:30am-12:35pm Pathogenesis of Bronchiectasis in Rare Genetic Diseases: Informing Potential Therapeutic Targets
     
Session Moderator: Margaret Leigh, MD

  • 10:30-10:50: Vicious Cycle of Airway Disease Leading to Bronchiectasis: Margaret Leigh, MD
    Live Q&A 10:50-10:55
  • 10:55-11:15: Mucous Rheology & Mucus Clearance: Ric Boucher, MD
    Live Q&A 11:15-11:20
  • 11:20-11:40: Airway Inflammation and Oxidative Stress: Jim Chmiel, MD
    Live Q&A 11:40-11:45
  • 11:45-12:05: Microbiome and Respiratory Infection: John Lipuma, MD, PhD
    Live Q&A 12:05-12:10
  • 12:10-12:30: Dysregulated Immunity (Innate and Adaptive): Steve Holland, MD
    Live Q&A 12:30-12:35

12:35-1:15pm Break

1:15-3:00pm Interventional Trials in Rare Genetic Causes of Bronchiectasis: Lessons Learned from Previous Trials in CF and Non-CF Bronchiectasis
     
Session Moderator: Peadar Noone, MD

  • 1:15-1:35: Key Lessons from Interventional Trials in CF: Bonnie Ramsey, MD
    Live Q&A 1:35-1:40
  • 1:40-2:00: Lessons from Interventional Trials in Non-CF Bronchiectasis: Ken Olivier, MD MPH
    Live Q&A 2:00-2:05
  • 2:05-2:25: Lessons from Trials Targeting inflammation: Scott Sagel, MD, PhD
    Live Q&A 2:25-2:30
  • 2:30-2:50: Lessons from Trials of Non-tuberculous Mycobacteria Treatment: Jerry Nick, MD
    Live Q&A 2:50-2:55

Day 2 - September 24, 2021:

8:00-9:00am Endpoints for Clinical Trials in Rare Genetic Causes of Bronchiectasis
     
Session Moderators: BreAnna Kinghorn, MD MS, Scott Sagel, MD PhD

  • 8:00-8:15: Lung function: Spirometry & Lung Clearance Index: Jessica Pittman, MD, MPH
  • 8:15-8:30: Pulmonary Exacerbations: Margaret Rosenfeld, MD
  • 8:30-8:45: Patient Reported Outcomes:  Sharon Dell, MD
  • 8:45-9:00: Live Q&A Panel Discussion with Drs Pittman, Rosenfeld, and Dell

9:00-10:00am Precision Medicine Clinical Trials
     
Session Moderator: Tom Ferkol, MD

  • 9:00-9:20: Molecular Therapies for PCD:  Heymut Omran, MD
    Live Q&A 9:20-9:25
  • 9:25-9:45: Gene Therapies for PIDs: David Rawlings, MD, PhD
    Live Q&A 9:45-9:50

9:50-10:05 Break

10:05-11:15 Developing Partnerships and Innovative Study Designs for Trials in Rare Diseases
     
Session Moderator: Margaret Rosenfeld, MD

  • 10:05-10:25: Partnering with Stakeholders: Foundations & Family: Michele Manion, Kathryn Stevens, Robert Sandhaus, MD, PhD
    Live Q&A 10:25-10:30
  • 10:30-10:50: The Role of Registries: Michael O’Conner, MD
    ​​​Live Q&A 10:50-10:55
  • 10:55-11:15: Designs for Small Sample Sizes: Nicole Hamblett, PhD
    Live Q&A 11:15-11:20
  • 11:20-11:40: Moving translational discoveries into clinical trials: Airway Mucins: Burton Dickey
    Live Q&A 11:40-11:45

11:45-12:15 Future Research Collaborations
      
Session Moderators: Stephanie Davis, MD, Margaret Rosenfeld, MD

  • 11:45-12:05: Panel Discussion: Future Research Collaborations: Steering Committee members, Robert Sandhaus, MD, PhD
  • 12:05-12:15: Wrap-Up & Closing Remarks: Tom Ferkol, MD, Stephanie Davis, MD, BreAnna Kinghorn, MD, MS, Margaret Rosenfeld, MD, MPH
Children's Hospital Colorado
Aurora, CO 80045
United States

This conference will be hosted virtually for all attendees. Detailed information will be provided within two weeks of the conference. This registration website will walk you through your expectations based on your attendee type.

Principal Investigators:

  • BreAnna Kinghorn, University of Washington, Seattle, Washington 
  • Margaret Rosenfeld MD MPH, University of Washington, Seattle, Washington 
  • Scott Sagel, MD PhD, University of Colorado, Denver, Colorado 
  • Adam Shapiro, MD, McGill University, Montreal, Quebec.

Steering Committee:

  • Stephanie Davis MD, University of North Carolina, Chapel Hill, North Carolina 
  • Margaret Leigh, University of North Carolina, Chapel Hill, North Carolina
  • Tom Ferkol MD, Washington University, St. Louis, Missouri
  • Steve Lommatzsch MD, National Jewish Health, Denver Colorado

Presenting Faculty:

  • Ric Boucher, MD
  • Jim Chmiel, MD
  •  Megan Cooper, MD
  •  Stephanie Davis, MD
  • Sharon Dell, MD
  • Christopher Evans, Ph.D.
  • Tom Ferkol, MD
  • Nicole Hamblett, PhD
  • Steve Holland, MD
  • Margaret Leigh, MD
  • John Lipuma, MD, PhD
  • Michele Manion
  • Jerry Nick, MD
  • Peadar Noone, MD
  • Michael O’Conner, MD
  • Jessica Pittman, MD, MPH
  • Deepika Polineni, MD 
  • Bonnie Ramsey, MD
  • Robert Sandhaus, MD, PhD
  • Kathryn Stephens

Medical:  Children’s Hospital Colorado is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians. 
Children’s Hospital Colorado designates this Other activity (Internet Live Activity) for a maximum of XX AMA PRA Category 1 Credit(s)™. Physicians should only claim credit commensurate with the extent of their participation in the activity.

General Attendance:  A general certificate of attendance will be available for all other care providers.

Available Credit

  • 8.75 AMA PRA Category 1 Credit™
  • 8.75 Attendance

Price

Cost:
$20.00
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*The mobile number feature is not enabled for international guests. International guests, please complete the registration process.

Required Hardware/software

Virtual participation in this conference will require a computer/tablet, with speakers and a microphone. For any questions or concerns, please contact us.