Pediatric CF Clinical Trials

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 April 11th, 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 March 7th, 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.

Contact our research team to learn more about participating in a pediatric cystic fibrosis clinical trial!