restarting triple therapy with robust monitoring for adverse events (retrial)

Conditions: Cystic Fibrosis

Summary: 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 drug-induced liver injury while taking elexacaftor/tezacaftor/ivacaftor (eti), start taking vanzacaftor/tezacaftor/deutivacaftor (vtd).

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) drug-induced liver injury attributed eti requiring dose modification or discontinuation (retrial-liver).

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.

Keywords: patient care adverse events monitoring