Restarting Triple Therapy With Robust Monitoring for Adverse Events (RETRIAL)
Start Date
5/1/2025
Completion Date
6/1/2028
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.
Detailed Description
RETRIAL is a prospective, longitudinal, observational, multi-site study of people with Cystic Fibrosis (PWCF) ages 6 and up who had to either change how they took or stop taking elexacaftor/tezacaftor/ivacaftor (ETI) due to new or worsening mental health/cognitive symptoms (such as depression, anxiety, mood, sleep, and/or brain fog/memory problems) and/or liver issues (elevated liver enzymes) while taking the standard dose, and who plan to start taking vanzacaftor/tezacaftor/deutivacaftor (VTD).
Eligibility Criteria
Age Range: 6 years to No maximum
Interventions
RETRIAL-Mental Health
RETRIAL-Liver
RETRIAL-Neuro
Conditions
Locations
University of Alabama at Birmingham
Birmingham, Alabama 35233
United States
Children's Hospital Colorado
Aurora, Colorado 80045
United States
National Jewish Health
Denver, Colorado 80206
United States
Nemours Children's
Wilmington, Delaware 19803
United States
Nemours Children's
Jacksonville, Florida 32207
United States
Nemours Children's
Orlando, Florida 32827
United States
Nemours Children's
Pensacola, Florida 32514
United States
Emory University
Atlanta, Georgia 30322
United States
Indiana University
Bloomington, Indiana 47405
United States
Kansas University Medical Center
Kansas City, Kansas 66160
United States
Johns Hopkins University
Baltimore, Maryland 21287
United States
Massachusetts General Hospital
Boston, Massachusetts 02114
United States
Boston Children's Hospital
Boston, Massachusetts 02215
United States
University of Michigan
Ann Arbor, Michigan 48109
United States
Washington University at St. Louis
St Louis, Missouri 63130
United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania 15224
United States
University of Texas Southwestern Medical Center
Dallas, Texas 75235
United States
Seattle Children's Hospital
Seattle, Washington 98105
United States