Recruiting

VTD

Sponsor:

Boston Children's Hospital

Code:

NCT06683092

Conditions

Cystic Fibrosis

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

Interventions

RETRIAL-Mental Health

RETRIAL-Liver

RETRIAL-Neuro

Study Details

Brief 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.

Conditions

Cystic Fibrosis

Study ID

NCT06683092

Start date

May 1, 2025

Status verified date

Oct, 2025

Completion date

Jun, 2028

Anticipated

Primary completion date

Mar, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

200 participants

Anticipated

Interventions and Outcome Measures

Arms

RETRIAL-Mental Health

People with CF ages 6 and up with a history of new or worsening mental health symptoms (such as depression, anxiety, mood, sleep) while on elexacaftor/tezacaftor/ivacaftor (ETI) requiring discontinuation or change from standard dosing who start taking vanzacaftor/tezacaftor/deutivacaftor (VTD).

RETRIAL-Neuro

People with CF from RETRIAL-Mental Health who experienced new/worsening neurocognitive symptoms (such as brain fog or memory problems) while taking elexacaftor/tezacaftor/ivacaftor (ETI).

RETRIAL-Liver

People with Cystic Fibrosis ages 6 and up with a history of drug-induced liver injury (such as elevated liver enzymes) attributed to elexacaftor/tezacaftor/ivacaftor (ETI) requiring dose modification or discontinuation who start taking vanzacaftor/tezacaftor/deutivacaftor (VTD).

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.

Primary outcome measure

  • RETRIAL-Mental Health (1st objective - discontinuation): one-year incidence of discontinuation or change from VTD standard dosing due to new/worsening mental health symptoms. [ Time Frame: Baseline to Month 12 ]
  • RETRIAL-Mental Health (2nd objective - symptoms): proportion of participants that experience any worsening in mental health symptoms during the 6-month follow-up [ Time Frame: Baseline to Month 6 ]
  • RETRIAL-Mental Health (3rd objective - RETRIAL-Neuro): proportion of participants that have a worsening on the NIH Toolbox's Fluid Cognition Composite score at 1 month follow-up. [ Time Frame: Baseline to Day 30 ]
  • RETRIAL-Liver: one-year incidence of drug-induced liver injury (DILI) with VTD, defined by expert consensus after review of clinical and laboratory data. [ Time Frame: Baseline to Month 12 ]

Central Contacts and Locations

Central contacts

Evelyn Bord, BS

617-919-6154evelyn.bord@childrens.harvard.edu

Callie Bacon, MPH

617-919-7696callie.bacon@childrens.harvard.edu

Locations

University of Alabama at Birmingham

Recruiting

Birmingham, Alabama, United States, 35233

Contacts

Kathryn Monroe

kathrynmonroe@uabmc.edu

Children's Hospital Colorado

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Allison Keck

allison.keck@childrenscolorado.org

Principal Investigator:

Emma Lyons, PhD

National Jewish Health

Recruiting

Denver, Colorado, United States, 80206

Contacts

Katie Poch

pochk@njhealth.org

Principal Investigator:

CJ Bathgate, PhD

Nemours Children's

Recruiting

Wilmington, Delaware, United States, 19803

Contacts

Kimmy Renner

kimberly.renner@nemours.org

Principal Investigator:

Kimberly Canter, PhD

Nemours Children's

Recruiting

Jacksonville, Florida, United States, 32207

Contacts

Jennifer Gafford

jennifer.gafford@nemours.org

Principal Investigator:

Kimberly Canter, PhD

Nemours Children's

Recruiting

Orlando, Florida, United States, 32827

Contacts

Sherry Wooldridge

sherry.wooldridge@nemours.org

Principal Investigator:

Kimberly Canter, PhD

Nemours Children's

Recruiting

Pensacola, Florida, United States, 32514

Contacts

Rebecca Davis

rebecca.davis@nemours.org

Principal Investigator:

Kimberly Canter, PhD

Emory University

Recruiting

Atlanta, Georgia, United States, 30322

Contacts

Ashleigh Streby

ashleigh.streby@emory.edu

Principal Investigator:

Kimberly Dickinson, MD

Indiana University

Recruiting

Bloomington, Indiana, United States, 47405

Contacts

Lisa Bendy

lbendy@iu.edu

Principal Investigator:

Emma Tillman, PhD PharmD

Kansas University Medical Center

Recruiting

Kansas City, Kansas, United States, 66160

Contacts

Megan Behrman

mbehrman@kumc.edu

Lawrence Scott

lscott2@kumc.edu

Principal Investigator:

Amanda Bruce, PhD

Johns Hopkins University

Recruiting

Baltimore, Maryland, United States, 21287

Contacts

Josephine Yodzis

jyodzis1@jh.edu

Principal Investigator:

Noah Lechtzin, MD

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02215

Contacts

Sara Carvalho

sara.carvalho@childrens.harvard.edu

Principal Investigator:

Carolyn Snell, PhD

University of Michigan

Recruiting

Ann Arbor, Michigan, United States, 48109

Contacts

Dawn Kruse

dmkruse@med.umich.edu

Principal Investigator:

Hanna Phan, PharmD

University of Pittsburgh Medical Center

Recruiting

Pittsburgh, Pennsylvania, United States, 15224

Contacts

Adrienne Dericco

adrienne.dericco2@upmc.edu

Principal Investigator:

Traci Kazmerski, MD

University of Texas Southwestern Medical Center

Recruiting

Dallas, Texas, United States, 75235

Contacts

Keianna Brown

Keianna.Brown@utsouthwestern.edu

Principal Investigator:

David Finklea, MD

Seattle Children's Hospital

Recruiting

Seattle, Washington, United States, 98105

Contacts

Gracia Bridges

gracia.bridges@seattlechildrens.org

Principal Investigator:

Freda Liu, PhD

More Information

Sponsor

Boston Children's Hospital

Last update posted

Nov 3, 2025

Last verified

Oct, 2025

Keywords

  • patient care
  • adverse events
  • monitoring

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Boston Children's Hospital on 2025-11-03.