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RECRUITING
NCT06683092

Restarting Triple Therapy With Robust Monitoring for Adverse Events (RETRIAL)

Sponsor: Boston Children's Hospital

View on ClinicalTrials.gov

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

OTHER

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).

OTHER

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.

OTHER

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