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New CF drug safety check: will VTD be safer for those who struggled with previous treatment?

NCT ID NCT06683092

Knowledge-focused Sponsor: Boston Children's Hospital Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study follows 200 people with cystic fibrosis (ages 6 and up) who had mental health or liver issues while taking the drug elexacaftor/tezacaftor/ivacaftor (ETI) and had to stop or change it. They will now start a newer triple therapy called vanzacaftor/tezacaftor/deutivacaftor (VTD). Researchers will track side effects through daily diaries, surveys, and liver tests for up to a year to see if VTD is better tolerated.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
vanzacaftor/tezacaftor/deutivacaftor (VTD)
What this could lead to
If successful, this study could help doctors understand how to safely use VTD in people with CF who have had mental health or liver problems from other treatments.
What could go wrong
This is an observational study, not a treatment trial. It will not test if VTD works, only monitor side effects. Results may not apply to all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 200 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2025

Expected to finish

Jun 2028

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Female and male people with CF ages 6 and older who are not able to tolerate the standard dose of ETI due to either new/worsening mental health symptoms and/or drug-induced liver injury.

Ages

6 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

cystic fibrosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Cystic fibrosis

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    18 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02215, United States

  • Children's Hospital Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Emory University

    RECRUITING

    Atlanta, Georgia, 30322, United States

  • Indiana University

    RECRUITING

    Bloomington, Indiana, 47405, United States

  • Johns Hopkins University

    RECRUITING

    Baltimore, Maryland, 21287, United States

  • Kansas University Medical Center

    RECRUITING

    Kansas City, Kansas, 66160, United States

  • Massachusetts General Hospital

    NOT_YET_RECRUITING

    Boston, Massachusetts, 02114, United States

  • National Jewish Health

    RECRUITING

    Denver, Colorado, 80206, United States

  • Nemours Children's

    RECRUITING

    Wilmington, Delaware, 19803, United States

  • Nemours Children's

    RECRUITING

    Jacksonville, Florida, 32207, United States

  • Nemours Children's

    RECRUITING

    Orlando, Florida, 32827, United States

  • Nemours Children's

    RECRUITING

    Pensacola, Florida, 32514, United States

  • Seattle Children's Hospital

    RECRUITING

    Seattle, Washington, 98105, United States

  • University of Alabama at Birmingham

    RECRUITING

    Birmingham, Alabama, 35233, United States

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • University of Pittsburgh Medical Center

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

  • University of Texas Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 75235, United States

  • Washington University at St. Louis

    NOT_YET_RECRUITING

    St Louis, Missouri, 63130, United States

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