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Recruiting NCT06683092

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

Trial Parameters

Condition Cystic Fibrosis
Sponsor Boston Children's Hospital
Study Type OBSERVATIONAL
Phase N/A
Enrollment 200
Sex ALL
Min Age 6 Years
Max Age N/A
Start Date 2025-05-01
Completion 2028-03
Interventions
RETRIAL-Mental HealthRETRIAL-LiverRETRIAL-Neuro

Brief Summary

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.

Eligibility Criteria

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: 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 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: 1. no m

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