Cystic Fibrosis (CF) Research Studies
The following studies are in progress at the University of Wisconsin Cystic Fibrosis Center in Madison, Wisconsin:
FIRST Study: Feeding Infants Right from the Start
- Inclusion criteria: Diagnosed with CF by newborn screening
- Purpose of the study: The purpose of the research is to understand how different types of infant feedings, breast milk and/or infant formula, affect growth and lung health in infants with cystic fibrosis (CF). This study concludes on the child’s second birthday.
FIRST Study: Feeding Infants Right from the Start- Phase 2
- Inclusion criteria: Infants who participated in the 0-2 years FIRST study.
- Purpose of the study: The purpose of the research is to understand how infant feeding and childhood food intake influences growth and lung health in children with CF over 2-6 years of age.
A Long-Term Prospective Observational Safety Study of the Incidence of and Risk Factors for Fibrosing Colonopathy in US Patients with Cystic Fibrosis Treated with Pancreatic Enzyme Replacement Therapy: A Harmonized Protocol Across Sponsors
- Inclusion criteria: Patients suspected of having fibrosing colonopathy (FC)
- Purpose of the study: The U.S. Food and Drug Administration (FDA) has asked the manufacturers of pancreatic enzyme supplements to study the incidence of FC in Cystic Fibrosis patients to see if there may be a link between taking pancreatic enzyme medications and developing FC.
MRI of Cystic Fibrosis Lung Disease Severity
- Inclusion criteria: Age ≥ 10 years of age
- Purpose of the study: The purpose of this study is to determine the ability of MRI to evaluate CF disease severity compared with the existing standards of breathing tests, chest x-ray, chest CT, and quality of life questionnaire.
The EPIC Observational Study: Longitudinal Assessment of Risk Factors For and Impact of Pseudomonas aeruginosa Acquisition and Early Anti-Pseudomonal Treatment in Children with CF
- Inclusion criteria: Previous participation in the EPIC observational study for years 6-10.
- Purpose of the study: The purpose of this study is to answer questions about Pseudomonas aeruginosa (Pa) and other germs that cause lung infections in people who have CF.
The effects of viral infections and antibiotics on the microbiome in CF
- Inclusion criteria: infants with CF who are less than 4 months of age
- Purpose of the study: The purpose of the research is to understand how viral infections and antibiotics affect the bacteria that reside in the upper airway and stool of children with CF, and if these effects are related to early CF lung disease.
ARRIVAL: A Phase 3 Study of Ivacaftor in Subjects less than 24 months with a CFTR Gating mutation.
- Inclusion criteria: Diagnosis of CF with a CFTR gating mutation, Age less than 24 months
- Purpose of the study: To evaluate the safety and tolerability of ivacaftor in subjects with CF who are <24 months of age at the time of treatment initiation and have a CFTR gating mutation on at least 1 allele
Standardized Treatment of Pulmonary Exacerbations II (STOP2)
- Inclusion criteria: >Age >17 years and plan to initiate IV antibiotics for a pulmonary exacerbation
- Purpose of the study: This randomized, controlled, open-label study is designed to evaluate the efficacy and safety of differing durations of IV treatment, given in the hospital or at home for a pulmonary exacerbation in adult patients with CF.
Phase 3 study of VX-445 triple combination drug in people with CF 12 years and older who have one copy of the F508del mutation and one copy of a minimal function mutation (Vertex VX17-445-102)
- Inclusion criteria: Diagnosis with CF (age ≥12 years), with one F508del mutation and a second “minimal function” mutation. A list of the eligible mutations can be found here: https://www.cff.org/PDF-Archive/Study-VX-445-102-Eligible-Mutations-April-2018.pdf
- Purpose of the study: To evaluate the effectiveness and safety of triple combination therapy of tezacaftor, ivacaftor and VX-445. Subjects will be randomly assigned to either placebo or the triple combination treatment and receive this for 24 weeks. After this 24 week study, subjects are eligible to roll over into an open label study in which everyone receives the triple combination therapy.
A Phase 3, 2-part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
- Inclusion criteria: Diagnosis of CF, age 1-2 years, with two copies of the F508del mutation.
- Purpose of the study: To evaluate the pharmacokinetics and safety of lumacaftor/ivacaftor in patients with CF who are 1-2 years of age.
CHEC-SC: Characterizing CFTR Modulated Changes in Sweat Chloride and their Association with Clinical Outcomes
- This study is examining the change in sweat chloride values in patients who are receiving CFTR modulator drugs (Kalydeco, Orkambi, or Symdeko). The study involves a single clinic visit with pulmonary function testing and a sweat test. Patients must have been receiving a modulator drug for at least 3 months.
If you have questions about these studies, please contact our CF research coordinators:
- Adult patients: Chiron Stevens, (608) 263-1244
- Pediatric patients: Linda Makholm, (608) 262-0340