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

ET

Emma Tillman

Principal Investigator

Status: Enrolling By Invitation Ages: 6 Years Gender: All Genders Phase: N/A 2 Locations

Brief Description

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

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

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.

Interested in participating?

Enrollment in IU School of Medicine clinical trials is managed through our All IN for Health program. Many studies only accept people who are part of an invited group. Visit allinforhealth.info to connect with the study team, or contact your doctor to see if you are eligible.