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

Launched by BOSTON CHILDREN'S HOSPITAL · Nov 8, 2024

Keywords

ClinConnect Summary

The RETRIAL study is looking at how people with Cystic Fibrosis (CF) can safely restart a new medication called vanzacaftor/tezacaftor/deutivacaftor (VTD) after experiencing mental health issues or liver problems while on another treatment called elexacaftor/tezacaftor/ivacaftor (ETI). This research is important because it aims to understand the effects of this new treatment while keeping a close watch on any side effects that may occur. The study is not yet recruiting participants but is open to individuals aged 6 and older who have had new or worsening mental health symptoms or liver toxicity after starting ETI.

To participate, individuals must be eligible for VTD and willing to complete some assessments before starting the new treatment. They will need access to a smartphone or tablet to receive text messages and must be English-speaking. If a child under 12 years old is participating, their primary caregiver will help by completing daily diaries about the child’s health. Overall, participants can expect to be closely monitored during the study to ensure their safety as they transition to this new medication.

Gender

ALL

Eligibility criteria

• 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 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 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 and intending to take it;
• * Experienced drug-induced liver injuary (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 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
• • Currently taking VTD
• • Unable or unwilling to follow protocol
• • If \<12 years old, having another \<12-year-old person in the same household also enrolled in the study
• • Is actively listed on any transplant list, or is less than 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 taking VTD
• • Unable or unwilling to follow protocol
• • If \<12 years old, having another \<12-year-old person in the same household also enrolled in the study
• • Any severe, decompensated liver disease (e.g. Child-Pugh, Class C)
• • Is actively listed on any transplant list, or is less than 3 moths post-transplant surgery (any organ), or had a 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.
• 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
• • 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.