Community Voice members have supported recent and ongoing work shaping the development of research on using modulators to treat the developing fetus with CF in people who do not have CF but carry a CF mutation (pregnant CF carriers). Read on to learn how parents of children with CF are informing study design and educational materials to advance scientific and patient understanding in this emerging space.
Focus Group Results Shaping Pregnancy and Modulator Dosing Study
Researchers from the University of Arkansas solicited input from the community on the design of their upcoming study that will monitor and evaluate the effects of Trikafta in pregnancy and the early neonatal period. The goal of the study is to inform appropriate Trikafta dosing for pregnant CF carriers to prevent CF-related complications in their developing child with CF. Data gathered through this study could support the development of “on-label” prescribing guidelines for Trikafta that would be covered by insurance.
The researchers hosted a focus group in January 2025 with women who are CF carriers thinking of becoming pregnant, currently pregnant, or parents of a young child with CF. Participants shared the challenges or barriers they may face when participating in such a study, their concerns about taking Trikafta during pregnancy, and openness to allowing their newborn to participate in the study. Read more about key themes and findings from the focus group here.
The research team used the feedback from the focus group to refine the study plan, including their approach to communications and outreach to parents who participate. The study is awaiting final review and funding approval. Thank you to the 10 Community Voice members who participated in the focus group!
Workshop Shaping Pregnancy and Modulator Dosing Study Updates
Last fall, we shared outcomes of the (PROTECT) Workshop, a day-long event hosted at CF Foundation headquarters for researchers, clinicians, and community members to discuss considerations for research and study design around prenatal use of modulators to prevent complications in babies with CF. Since then, two working groups have formed to advance efforts discussed during the workshop:
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The Study Design Working Group aims to design an observational study to learn whether CF carriers can safely take highly effective modulators during pregnancy to treat or prevent CF complications such as meconium ileus, pancreatic insufficiency, absence of the vas deferens, and delayed growth in their child with CF. The study will also assess the safety and efficacy of modulators in the infant with CF. We’ll share more information about this study in late 2025.
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Resources Working Group is developing educational materials for community members and providers to improve awareness of the possibility that CF carriers might take modulators while pregnant with or breastfeeding a child with CF. Materials will address what specific providers, such as genetic counselors and maternal-fetal medicine specialists, need to know about CF and modulators, and will include templates for community members to use when seeking approval from their insurance companies for modulator use during pregnancy.
We look forward to sharing more updates on this work, including highlights shared at the North American Cystic Fibrosis Conference (NACFC) in October!
