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

Prior treatment

• Currently, or prior history of, taking VTD
• Is actively listed on any transplant list, or within 3 months post-transplant surgery
• Currently, or prior history of, taking VTD

Other illness

• 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

Age

• If \<12 years old, having another \<12-year-old person in the same household consented into the study
• If \<12 years old, having another \<12-year-old person in the same household consented into the study

Other

• RETRIAL-Mental Health:
• Cannot access VTD
• Unable or unwilling to follow protocol
• 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
• Unable or unwilling to follow protocol
• 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:
• \- Any situation that, in the opinion of the Investigator, would compromise the safety of the patient or the quality of the data.

• 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.
• 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.