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

Status: Recruiting
Location: See all (18) locations...
Intervention Type: Other
Study Type: Observational
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
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: f
View:

∙ 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.

Locations
United States
Alabama
University of Alabama at Birmingham
RECRUITING
Birmingham
Colorado
Children's Hospital Colorado
RECRUITING
Aurora
National Jewish Health
RECRUITING
Denver
Delaware
Nemours Children's
RECRUITING
Wilmington
Florida
Nemours Children's
RECRUITING
Jacksonville
Nemours Children's
RECRUITING
Orlando
Nemours Children's
RECRUITING
Pensacola
Georgia
Emory University
RECRUITING
Atlanta
Indiana
Indiana University
RECRUITING
Bloomington
Kansas
Kansas University Medical Center
RECRUITING
Kansas City
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
Massachusetts General Hospital
NOT_YET_RECRUITING
Boston
Maryland
Johns Hopkins University
RECRUITING
Baltimore
Michigan
University of Michigan
RECRUITING
Ann Arbor
Missouri
Washington University at St. Louis
NOT_YET_RECRUITING
St Louis
Pennsylvania
University of Pittsburgh Medical Center
RECRUITING
Pittsburgh
Texas
University of Texas Southwestern Medical Center
RECRUITING
Dallas
Washington
Seattle Children's Hospital
RECRUITING
Seattle
Contact Information
Primary
Evelyn Bord, BS
evelyn.bord@childrens.harvard.edu
617-919-6154
Backup
Callie Bacon, MPH
callie.bacon@childrens.harvard.edu
617-919-7696
Time Frame
Start Date: 2025-05-01
Estimated Completion Date: 2028-06
Participants
Target number of participants: 200
Treatments
RETRIAL-Mental Health
People with CF ages 6 and up with a history of new or worsening mental health symptoms (such as depression, anxiety, mood, sleep) while on elexacaftor/tezacaftor/ivacaftor (ETI) requiring discontinuation or change from standard dosing who start taking vanzacaftor/tezacaftor/deutivacaftor (VTD).
RETRIAL-Neuro
People with CF from RETRIAL-Mental Health who experienced new/worsening neurocognitive symptoms (such as brain fog or memory problems) while taking elexacaftor/tezacaftor/ivacaftor (ETI).
RETRIAL-Liver
People with Cystic Fibrosis ages 6 and up with a history of drug-induced liver injury (such as elevated liver enzymes) attributed to elexacaftor/tezacaftor/ivacaftor (ETI) requiring dose modification or discontinuation who start taking vanzacaftor/tezacaftor/deutivacaftor (VTD).
Related Therapeutic Areas
Sponsors
Collaborators: Indiana University, Washington University School of Medicine, University of Kansas Medical Center, Children's Hospital Colorado, Cystic Fibrosis Foundation, Massachusetts General Hospital, National Jewish Health, Johns Hopkins University
Leads: Boston Children's Hospital

This content was sourced from clinicaltrials.gov