Upcoming & Enrolling Pediatric Cystic Fibrosis Clinical Trials
The studies listed below will soon be or are currently enrolling participants. Our list of upcoming studies is constantly evolving. Please contact our research team to learn about clinical trials in our pipeline that may not be listed here!
To learn more about upcoming clinical trials in CF, visit the Cystic Fibrosis Foundation’s Clinical Trials Finder.
Last updated May 9th, 2024.
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CHEC-OB-17 | Sweat Chloride Collection
“This study is taking place at multiple care centers across the U.S. It will look at sweat chloride concentration in people who are currently taking CFTR modulators.” – CFF Trial Finder Profile
Principal Investigator: Hara Levy, MD
Lead Research Coordinator: Melanie Nelson
Key Inclusion Criteria:
– Enrolled in CFF Patient Registry
– On current modulator for > 90 days
Time Commitment: One time sweat test.
See CFF Trial Finder profile here.
See ClinicalTrials.gov profile here.
CF Family Biobank | Observational Questionnaire & Blood Banking Study
We are seeking children and young adults (0-25 years old) with a diagnosis of cystic fibrosis (CF) or a CF-related disorder, along with their immediate family members, to join a research study. Parents and siblings are welcome and encouraged to join and help us learn about how CF impacts children and their families. Everyone who participates will give a blood sample and complete a health questionnaire.
Principal Investigator: Hara Levy, MD
Key Inclusion Criteria:
– 0-25 years old with a diagnosis of CF or a CF-related disorder
– Biological parents and siblings of a person with CF or a CF-related disorder participating in this study
Time Commitment:
– All: 1 initial visit
– Children with CF and their siblings: 4 additional optional visits over 3 years
Ongoing Pediatric Cystic Fibrosis Clinical Trials
The clinical trials listed below are currently ongoing and are no longer accepting new participants. Our studies are both investigational and observational. We enjoy working with families from all around the Midwest. Please contact our research team to learn about clinical trials in our pipeline that may not be listed here!
To learn more about upcoming clinical trials in CF, visit the Cystic Fibrosis Foundation’s Clinical Trials Finder.
Last updated May 9th, 2024.
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BEGIN-OB-19 Part B | Observing CFTR modulators in infants and young children
“This two-part observational study will look at the effects of CFTR modulators on growth in young children with CF. These drugs are intended to help CFTR protein function closer to normal.” – CFF Trial Finder Profile
Principal Investigator: Christina Barreda, MD
Lead Research Coordinator: Melanie Nelson
See CFF Trial Finder profile here.
See ClinicalTrials.gov profile here.
PROMISE-OB-18 Pediatric Cohort | Observational Trikafta Study
“This observational study will measure the effects of the triple-combination therapy, elexacaftor/tezacaftor/ivacaftor, in children with CF. These drugs are intended to help CFTR protein function closer to normal. This study will look at how treatment with the triple-combination therapy affects children with CF across many different aspects of the disease.” – CFF Trial Finder Profile
Principal Investigator: Andrew Braun, MD
Lead Research Coordinator: Nikki Cole
See CFF Trial Finder profile here.
See ClinicalTrials.gov profile here.
VX21-121-105 | Once a Day New Modulator
“The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.” – ClinicalTrials.gov Profile
Principal Investigator: Hara Levy, MD
Lead Research Coordinator: Megan Devereaux
See ClinicalTrials.gov profile here.
VX22-121-106 | Open Label Once a Day New Modulator
“The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).” – ClinicalTrials.gov Profile
Principal Investigator: Hara Levy, MD
Lead Research Coordinator: Megan Devereaux
See ClinicalTrials.gov profile here.