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NCT06683092Recruiting

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

Boston Children's Hospital

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

Inclusion Criteria: RETRIAL-Mental Health: * PWCF age 6 years and up (if age is \< 12 years old, the PWCF's caregiver will complete daily diaries and surveys; see "Caregiver Participant Inclusion Criteria" below) * Eligible for VTD and intending to take it * Experienced new or worsening mental health symptoms after initiating ETI, which led to one of the following changes in treatment to currently taking: 1. No modulators 2. A modulator other than ETI 3. A flipped dose of ETI 4. A reduced dose of ETI * Willing to delay first VTD dose for short period of time to complete the Baseline assessments * Has access to a smart device (phone, tablet, etc.) capable of receiving messages with survey links * Is English-speaking. RETRIAL-LIVER: * A person with CF age 6 years and up * Eligible for VTD and intending to take it * Experienced drug-induced liver injury (as defined by local care team) after initiating ETI, which led to one of the following changes in treatment to currently taking: 1. no modulators; or 2. a modulator other than ETI; or 3. a reduced or altered dose of ETI; * Willing to delay first VTD dose for short period of time to complete the Baseline assessments * Has access to a smart device (phone, tablet, etc.) capable of receiving messages with survey links * Is English-speaking. Exclusion Criteria: RETRIAL-Mental Health: * Cannot access VTD * Currently, or prior history of, taking VTD * Unable or unwilling to follow protocol * If \<12 years old, having another \<12-year-old person in the same household consented into the study * Is actively listed on any transplant list, or within 3 months post-transplant surgery * Is currently pregnant (test not required) * Anticipated change in CF Care Centers in the next 6 months * Any situation that, in the opinion of the Investigator, would compromise the safety of the patient or the quality of the data. RETRIAL-LIVER: * Cannot access VTD * Currently, or prior history of, taking VTD * Unable or unwilling to follow protocol * If \<12 years old, having another \<12-year-old person in the same household consented into the study * Any severe, decompensated liver disease (e.g. Child-Pugh, Class C) * Is actively listed on any transplant list, or within 3 months post-transplant surgery (any organ), or history of liver transplant * Is currently pregnant (test not required) * Anticipated change in CF Care Centers in the next 6 months * Any situation that, in the opinion of the Investigator, would compromise the safety of the patient or the quality of the data. Caregiver Participant: Inclusion Criteria: * Is a primary, daily caregiver of a person with CF under the age of 12 enrolled in the study * Has access to smart device (phone, tablet, etc.) capable of receiving messages with survey links * Is able to read and complete surveys and Daily Diary in English. Exclusion Criteria: \- Any situation that, in the opinion of the Investigator, would compromise the safety of the patient or the quality of the data.

Interventions

OTHER

RETRIAL-Mental Health

OTHER

RETRIAL-Liver

OTHER

RETRIAL-Neuro

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Conditions

Cystic Fibrosis

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