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Restarting Triple Therapy With Robust Monitoring for Adverse Events (RETRIAL)
Sponsor: Boston Children's Hospital
Summary
RETRIAL is a multi-site observational study of people with Cystic Fibrosis (PWCF) ages 6 and up starting the new triple-therapy modulator (vanzacaftor/tezacaftor/deutivacaftor (VTD)), after having experienced neuropsychiatric events and/or liver injury while taking elexacaftor/tezacaftor/ivacaftor (ETI) that resulted in a modification or discontinuation of standard ETI dosing.
Key Details
Gender
All
Age Range
6 Years - Any
Study Type
OBSERVATIONAL
Enrollment
200
Start Date
2025-05-01
Completion Date
2028-06
Last Updated
2025-11-03
Healthy Volunteers
No
Conditions
Interventions
RETRIAL-Mental Health
Participants will complete: daily diaries for 6 weeks, from about 2 weeks prior to starting VTD to about 4 weeks after; biweekly surveys, from about 2 weeks prior to starting VTD to 6 months after, and 2 quarterly surveys (at 9mo and 12mo post-initiation of VTD).
RETRIAL-Liver
Participants may have a research liver function test done prior to starting VTD and 28 days after, if not done clinically. Participants will complete surveys: before starting VTD, a month after, and quarterly (at about 3, 6, 9, and 12 months) following VTD initiation.
RETRIAL-Neuro
Participants will complete a neurocognitive assessment prior to starting VTD and 28 days after.
Locations (18)
University of Alabama at Birmingham
Birmingham, Alabama, United States
Children's Hospital Colorado
Aurora, Colorado, United States
National Jewish Health
Denver, Colorado, United States
Nemours Children's
Wilmington, Delaware, United States
Nemours Children's
Jacksonville, Florida, United States
Nemours Children's
Orlando, Florida, United States
Nemours Children's
Pensacola, Florida, United States
Emory University
Atlanta, Georgia, United States
Indiana University
Bloomington, Indiana, United States
Kansas University Medical Center
Kansas City, Kansas, United States
Johns Hopkins University
Baltimore, Maryland, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Washington University at St. Louis
St Louis, Missouri, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States