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

Cystic Fibrosis and Investigational Medication Study

E
Emma Tillman

Primary Investigator

Enrolling By Invitation
6 years or above
All
Phase N/A
10 participants needed
2 Locations

Brief description of study

RETRIAL is a prospective, longitudinal, observational, multi-site study designed to observe what happens when people with Cystic Fibrosis (PWCF) with a history new/worsening mental health symptoms or liver toxicity while taking elexacaftor/tezacaftor/ivacaftor (ETI), start taking vanzacaftor/tezacaftor/deutivacaftor (VTD).

THIS STUDY IS ENROLLING BY INVITATION ONLY - Eligible PWCF will be identified by CF care teams and research coordinators (RCs) at CF clinics of 15 participating sites through a review of patient medical records for inclusion/exclusion criteria. All PWCF who meet basic eligibility criteria can be approached or contacted by clinical staff or an RC for interest and further screening. The site investigator and/or site RC will provide PWCF and their families with an explanation of the study.

Detailed description of study

RETRIAL is a prospective, longitudinal, observational, multi-site study designed to observe what happens when people with Cystic Fibrosis (PWCF) ages 6 and up start taking vanzacaftor/tezacaftor/deutivacaftor (VTD) and have a history of (1) new or worsening mental health symptoms (MH) while on elexacaftor/tezacaftor/ivacaftor (ETI) requiring discontinuation or change from standard dosing (RETRIAL-MH), OR (2) liver-related intolerance to ETI requiring dose modification or discontinuation (RETRIAL-Liver).

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Cystic Fibrosis
  • Age: 6 years or above
  • Gender: All

Inclusion Criteria:

RETRIAL-MH:

  • 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
  • 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 triple dose for short period of time to complete the Baseline assessments
  • Has access to a smart device (phone, tablet, etc.) capable of receiving text messages
  • Is English-speaking.

RETRIAL-LIVER:

  • A person with CF age 6 years and up;
  • Eligible for VTD;
  • Experienced liver toxicity (as defined by local care team) after initiating ETI, which led to one of the following changes in treatment: currently
    1. taking no modulators; or
    2. taking a modulator other than ETI; or
    3. taking a reduced or altered dose of ETI;
  • Willing to delay first VTD triple dose for short period of time to complete the Baseline assessments;
  • Has access to a smart device (phone, tablet, etc.) capable of receiving text messages;
  • Is English-speaking.

Exclusion Criteria:

RETRIAL-MH:

  • Cannot access VTD
  • Unable or unwilling to follow protocol
  • Having a person in the same household who is also enrolled in the study (only for <18 years)
  • Is actively listed on any transplant list, or is less than 3 months post-transplant surgery
  • Is currently pregnant (test not required)
  • Any situation, in the opinion of the Investigator, would compromise the safety of the patient or the quality of the data.

RETRIAL-LIVER:

  • Cannot access VTD;
  • Unable or unwilling to follow protocol;
  • Having a person in the same household who is also enrolled in the study (only for <18 years);
  • Any person with other known liver disease (non-CF related);
  • Is actively listed on any transplant list or had a liver transplant;
  • Is currently pregnant (test not required);
  • Any situation, in the opinion of the Investigator, would compromise the safety of the patient or the quality of the data.

RETRIAL-MH 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 text messages;
  • Can complete the Daily Diary on behalf of the child for a total of 42 days (from 14 days pre-VTD to 28 days post-starting VTD);
  • Is English-speaking.

Exclusion Criteria:

- Any situation, in the opinion of the Investigator, would compromise the safety of the patient or the quality of the data.

This study investigates what happens when people with Cystic Fibrosis (PWCF) who have experienced mental health or liver-related issues with a previous medication start taking an investigational medication. Cystic Fibrosis is a condition that affects the lungs and digestive system, caused by a defective gene that leads to thick, sticky mucus. This study will observe the effects on those who had to stop or change their previous medication due to mental health symptoms or liver problems.

Participants will be observed as they begin taking the investigational medication. The study will involve monitoring for any changes or side effects. This is an observational study, which means that researchers will collect data by observing participants without changing their treatment. This helps to understand how the investigational medication works in people with specific conditions.

  • Who can participate: Children and adults aged 6 and older with Cystic Fibrosis who had mental health or liver issues from a previous treatment can join. Participants need to speak English and use a smart device.
  • Study details: Participants must delay starting the investigational medication briefly for initial checks and use a smart device for communication. Caregivers of children under 12 will complete daily diaries if needed.
Updated on 16 Oct 2025. Study ID: PPUL-BCH-RETRIAL, 26611
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Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting Referrals by Invitation Only