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Workshop

W25--Cutting-Edge Research: What's New?

Friday, September 27, 2024
10:15 AM – 12:15 PM ET
Location:210 A-C

Recorded Session

This session aims to report the results of the latest, high-impact clinical research conducted for the monitoring and management of cystic fibrosis (CF) in the highly-effective modulator era. The sessions speakers will discuss the results of the Vanzacaftor/Tezacaftor/Deutivacaftor Phase 3 trial and clinical effectiveness of Elexacaftor/Tezacaftor/Ivacaftor (ETI) on CF inflammation. The highly anticipated remote clinical trial data from HERO-2 and OUTREACH studies will be reviewed. Finally, we will assess the novel quantitative assay for ETI levels recently introduced for clinical use.

Learning Objectives:

  • Discuss the clinical efficacy and effectiveness of the latest highly effective modulator therapies in people with cystic fibrosis.
  • Evaluate the results of multicenter decentralized study designs and remote clinical endpoints.
  • Assess the novel quantitative assay for clinical measurement of elexacaftor/tezacafor/ivacaftor.

Presentations:

  • 10:15 AM – 12:15 PM ET
    W25.1- Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ Triple) in Adolescents and Adults with CF: Results from Two Randomized, Active-Controlled Phase 3 Trials

    Speaker: Claire Keating, MD – Columbia University Irving Medical Center

  • 10:15 AM – 12:15 PM ET
    W25.2- Quantification of Elexacaftor/Tezacaftor/Ivacaftor levels: Comparison between the STEADY Study validation cohort and real world experience

    Speaker: Silvia Caceres – National Jewish Health

  • 10:15 AM – 12:15 PM ET
    W25.3- Long-term effects of elexacaftor/tezacaftor/ivacaftor therapy on airway and systemic inflammation in CF

    Speaker: Scott Sagel, MD, PhD – Children's Hospital Colorado/University of Colorado Anschutz Medical Campus

  • 10:15 AM – 12:15 PM ET
    W25.4- Lung Function and Pulmonary Exacerbations in Individuals who Discontinue Chronic Daily Respiratory Therapies: Results of the HERO-2 Study

    Speaker: Cynthia Brown, MD, MS (she/her/hers) – Indiana University

  • 10:15 AM – 12:15 PM ET
    W25.5- Home Spirometry as a CF Clinical Trial Endpoint: Results of the OUTREACH Study

    Speaker: Margaret Rosenfeld, MD, MPH (she/her/hers) – University of Washington