Restarting Triple Therapy With Robust Monitoring for Adverse Events (RETRIAL)
Recruiting now
Conditions studied: Cystic Fibrosis
In brief
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 facts
- Study ID
- NCT06683092
- Run by
- Boston Children's Hospital
- People needed
- 200
- Starts
- 2025-05-01
- Expected to finish
- 2028-06-01
- Last updated by the study team
- 2025-11-03
Who can join
Age: 6 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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:
- 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.
You may not qualify if…
- 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:
Where it is running
- Seattle Children's Hospital — Seattle, Washington, United States (enrolling)
- Children's Hospital Colorado — Aurora, Colorado, United States (enrolling)
- National Jewish Health — Denver, Colorado, United States (enrolling)
- Nemours Children's — Wilmington, Delaware, United States (enrolling)
- Nemours Children's — Jacksonville, Florida, United States (enrolling)
- Nemours Children's — Orlando, Florida, United States (enrolling)
- Nemours Children's — Pensacola, Florida, United States (enrolling)
- Emory University — Atlanta, Georgia, United States (enrolling)
- Indiana University — Bloomington, Indiana, United States (enrolling)
- Kansas University Medical Center — Kansas City, Kansas, United States (enrolling)
- Johns Hopkins University — Baltimore, Maryland, United States (enrolling)
- University of Texas Southwestern Medical Center — Dallas, Texas, United States (enrolling)
- University of Alabama at Birmingham — Birmingham, Alabama, United States (enrolling)
- Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
- University of Michigan — Ann Arbor, Michigan, United States (enrolling)
- University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States (enrolling)
- Massachusetts General Hospital — Boston, Massachusetts, United States
- Washington University at St. Louis — St Louis, Missouri, United States
Full record on ClinicalTrials.gov
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