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Observational Study of Vanzacaftor/Tezacaftor/Deutivacaftor (VTD) in Cystic Fibrosis

This observational study, called RETRIAL, is for people with Cystic Fibrosis (CF) aged 6 and older. It looks at what happens when you start a new triple-therapy medicine called vanzacaftor/tezacaftor/deutivacaftor (VTD). This study is for those who previously had to change or stop taking elexacaftor/tezacaftor/ivacaftor (ETI) because of new or worsening mental health symptoms (like depression, anxiety, or brain fog) or liver problems. Researchers want to see how often people need to stop or change their VTD dose due to mental health issues, how many experience worsening mental health symptoms, and if there are changes in thinking abilities. You will complete surveys and some assessments over about a year.

Study design
This is an observational study involving about 200 participants with Cystic Fibrosis. It tracks participants over time without assigning them to different treatment groups.
What's involved
You will complete daily diaries for 6 weeks, biweekly surveys for 6 months, and quarterly surveys for a year. You may also have a research liver function test and a neurocognitive assessment.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for up to 12 months after starting VTD to monitor mental health symptoms and other outcomes.

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NCT06683092

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

Recruiting
Not specifiedAges 6+Observational
Boston Children's Hospital
~200 participants
Updated 2025-11-03 on ClinicalTrials.gov
What's tested:RETRIAL-Mental HealthRETRIAL-LiverRETRIAL-Neuro

At a glance

Recruiting sites
16 of 18 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
RETRIAL-Mental Health (1st objective - discontinuation): one-year incidence of discontinuation or change from VTD standard dosing due to new/worsening mental health symptoms.
Measured over Baseline to Month 12
+3 more outcomes measured

Conditions

Cystic Fibrosis

Explore related trials

Cystic Fibrosis

Where it's being run

18 sites across 14 states
Florida3
Colorado2
Massachusetts2
Alabama1
Delaware1
Georgia1
Indiana1
Kansas1

Study leadership

  • Anna Georgiopoulos, MD · PRINCIPAL_INVESTIGATOR · Massachusetts General Hospital
  • CJ Bathgate, PhD · PRINCIPAL_INVESTIGATOR · National Jewish Health
  • Janis Stoll, MD · PRINCIPAL_INVESTIGATOR · Washington University at St. Louis

Who to contact

Evelyn Bord, BS
[email protected]
617-919-6154
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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:
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.
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:
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

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.
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.

What this trial measures

  • RETRIAL-Mental Health (1st objective - discontinuation): one-year incidence of discontinuation or change from VTD standard dosing due to new/worsening mental health symptoms.Baseline to Month 12

    Discontinuation or change from VTD standard dosing due to mental health symptoms at any time during the one-year follow-up as reported in either: daily diaries, biweekly or quarterly surveys, or chart review.

  • RETRIAL-Mental Health (2nd objective - symptoms): proportion of participants that experience any worsening in mental health symptoms during the 6-month follow-upBaseline to Month 6

    Change from baseline that exceeds the minimally-important difference (MID) / minimally-important change (MIC) within the first 6 months after starting VTD in any of the following patient-reported outcome measures: (1) depression (PHQ-8 or PROMIS depression parent proxy), (2) anxiety (GAD-7 or PROMIS anxiety parent proxy), (3) anger and irritability (PROMIS-anger or PROMIS-anger parent proxy), (4) cognitive functioning (PROMIS- cognitive function or PROMIS-cognitive function parent proxy), (5) sleep disturbance (PROMIS-sleep disturbance or PROMIS-sleep disturbance parent proxy).

  • 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.Baseline to Day 30

    Decrease in age-adjusted standard score from baseline to 1 month on the NIH Toolbox's Fluid Cognition Composite Score (a performance-based neurocognitive measure) that exceeds MIC ≥ 7.5 points, signifying a worsening in function.

  • RETRIAL-Liver: one-year incidence of drug-induced liver injury (DILI) with VTD, defined by expert consensus after review of clinical and laboratory data.Baseline to Month 12

    Positive DILI diagnosis, including those characterized as "likely," "probable," and "highly probable," as reviewed by a hepatology panel of three experts who assign these likelihood scores based upon review of case report forms, including a specific clinical narrative and laboratory values assessing for other potential etiologies.