Overview
RETRIAL is a multi-site observational study of people with Cystic Fibrosis (PWCF) ages 6 and up starting the new triple-therapy modulator (vanzacaftor/tezacaftor/deutivacaftor (VTD)), after having experienced neuropsychiatric events and/or liver injury while taking elexacaftor/tezacaftor/ivacaftor (ETI) that resulted in a modification or discontinuation of standard ETI dosing.
Description
RETRIAL is a prospective, longitudinal, observational, multi-site study of people with Cystic Fibrosis (PWCF) ages 6 and up who had to either change how they took or stop taking elexacaftor/tezacaftor/ivacaftor (ETI) due to new or worsening mental health/cognitive symptoms (such as depression, anxiety, mood, sleep, and/or brain fog/memory problems) and/or liver issues (elevated liver enzymes) while taking the standard dose, and who plan to start taking vanzacaftor/tezacaftor/deutivacaftor (VTD).
Eligibility
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:
- No modulators
- A modulator other than ETI
- A flipped dose of ETI
- 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:
- no modulators; or
- a modulator other than ETI; or
- 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.