Restarting Triple Therapy With Robust Monitoring for Adverse Events (RETRIAL)
Boston Children's Hospital
200 participants
May 1, 2025
OBSERVATIONAL
Conditions
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.
Eligibility
Inclusion Criteria23
- RETRIAL-Mental Health:
- 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:
- No modulators
- A modulator other than ETI
- A flipped dose of ETI
- 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:
- no modulators; or
- a modulator other than ETI; or
- 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.
- 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 Criteria21
- 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:
- \- Any situation that, in the opinion of the Investigator, would compromise the safety of the patient or the quality of the data.
Interested in this trial?
Get notified about updates and connect with the research team.
Interventions
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).
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.
Participants will complete a neurocognitive assessment prior to starting VTD and 28 days after.
Locations(18)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06683092