Cystic Fibrosis Clinical Trials

NACFC 2023

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Once again, this year our research team traveled to Phoenix, AX for the 2023 North American Cystic Fibrosis Conference. At the conference specific research sessions included topics of  “What’s New and Updates in CF Research”, “Best practices and Innovative approaches for conducting trials”

and we heard from other centers’ experiences with special cases. While every year the plenaries offer great insight into overall Cystic Fibrosis care, we were excited how much the iconic sessions weighed in on research, specifically research that is happening right here at our center. Below are some highlights from each session and points that relate to work being done at UW. Videos are located at the bottom of this post or a link to all the sessions can be found here. 

Plenary 1: Genetic Therapies for All: Harnessing Cross-Disease Knowledge for Breakthroughs in CF 

  • The CF Foundation’s current Genetic Therapy Pipeline displays that studies range from the preclinical planning phase to phase 2, an active enrolling phase with CF patients. VX-522, a clinical trial happening at our center. 
  • Genetic therapies are currently and successfully used for sickle cell disease patients.  
  • It’s important to know the different types of gene therapies– Gene addition delivers new healthy CFTR gene at the cellular level, this method is reflected in the VX-522 study. Gene editing fixes a patient’s existing CFTR gene.  
  • There are multiple ways to deliver gene therapy at the cellular level, mRNA is how the CFTR gene is delivered in the VX-522 clinical trial.

Plenary 2: Micro-management: the Changing Face of Infections in CF 

  • Studies at our center like PROMISE-OB-18 have shown a decrease in average sputum densities of all pathogens examined from initiation of Trikafta 
  • The STOP (Standardizing Treatment Of Pulmonary Exacerbation) Program, a series of observational trials centered around treatment of pulmonary exacerbations that our site’s patients have been a part of lead to finding:
    • Antibiotic treatment for 10 days was not worse than treatment for 14 days
    • Antibiotic treatment for 21 days was not better than treatment for 14 days
  • The next impact the STOP program, STOP360, is investigating is the difference between treating Pulmonary Exacerbations with 1 vs 2 IV antibiotics and the difference between receiving IV antibiotics at home vs inpatient. 

Plenary 3: There is No Health Without Mental-Health: Progress, Challenges, & Hope for the Future

  • Just like the research study pipeline, there is a mental health pipeline for psychological interventions. The status’ of these treatments range from the developmental stage to the implementation phases in centers across the country. CF-CBT is an intervention occurring with our team’s mental Health Coordinator, Megan Karls.
  • TDN has developed PRIME, Prioritizing Research in Metal Health, a working group aligned with the Mental Health Advisory Committee with a mission to advance our knowledge of mental health conditions in the CF population throughout the lifespan and promote clinical research that will improve the prevention, diagnosis, and treatment of mental health complications of CF

See you next year in Boston!