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

NCT ID: NCT06683092

Last Updated: 2025-11-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Total Enrollment

200 participants

Study Classification

OBSERVATIONAL

Study Start Date

2025-05-01

Study Completion Date

2028-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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).

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cystic Fibrosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

OTHER

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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-Mental Health

Intervention Type OTHER

Participants will complete: daily diaries for 6 weeks, from about 2 weeks prior to starting VTD to about 4 weeks after; biweekly surveys, from about 2 weeks prior to starting VTD to 6 months after, and 2 quarterly surveys (at 9mo and 12mo post-initiation of 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-Neuro

Intervention Type OTHER

Participants will complete a neurocognitive assessment prior to starting VTD and 28 days after.

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).

RETRIAL-Liver

Intervention Type OTHER

Participants may have a research liver function test done prior to starting VTD and 28 days after, if not done clinically.

Participants will complete surveys: before starting VTD, a month after, and quarterly (at about 3, 6, 9, and 12 months) following VTD initiation.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

RETRIAL-Mental Health

Participants will complete: daily diaries for 6 weeks, from about 2 weeks prior to starting VTD to about 4 weeks after; biweekly surveys, from about 2 weeks prior to starting VTD to 6 months after, and 2 quarterly surveys (at 9mo and 12mo post-initiation of VTD).

Intervention Type OTHER

RETRIAL-Liver

Participants may have a research liver function test done prior to starting VTD and 28 days after, if not done clinically.

Participants will complete surveys: before starting VTD, a month after, and quarterly (at about 3, 6, 9, and 12 months) following VTD initiation.

Intervention Type OTHER

RETRIAL-Neuro

Participants will complete a neurocognitive assessment prior to starting VTD and 28 days after.

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

RETRIAL-Mental Health:

* 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 modulators; or
2. a modulator other than ETI; or
3. 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.

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:


\- Any situation that, in the opinion of the Investigator, would compromise the safety of the patient or the quality of the data.
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Cystic Fibrosis Foundation

OTHER

Sponsor Role collaborator

Johns Hopkins University

OTHER

Sponsor Role collaborator

Massachusetts General Hospital

OTHER

Sponsor Role collaborator

National Jewish Health

OTHER

Sponsor Role collaborator

Indiana University

OTHER

Sponsor Role collaborator

Children's Hospital Colorado

OTHER

Sponsor Role collaborator

University of Kansas Medical Center

OTHER

Sponsor Role collaborator

Washington University School of Medicine

OTHER

Sponsor Role collaborator

Boston Children's Hospital

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Gregory Sawicki

Co-Chair of Success with Therapies Research Consortium

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Anna Georgiopoulos, MD

Role: PRINCIPAL_INVESTIGATOR

Massachusetts General Hospital

CJ Bathgate, PhD

Role: PRINCIPAL_INVESTIGATOR

National Jewish Health

Janis Stoll, MD

Role: PRINCIPAL_INVESTIGATOR

Washington University at St. Louis

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status RECRUITING

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status RECRUITING

National Jewish Health

Denver, Colorado, United States

Site Status RECRUITING

Nemours Children's

Wilmington, Delaware, United States

Site Status RECRUITING

Nemours Children's

Jacksonville, Florida, United States

Site Status RECRUITING

Nemours Children's

Orlando, Florida, United States

Site Status RECRUITING

Nemours Children's

Pensacola, Florida, United States

Site Status RECRUITING

Emory University

Atlanta, Georgia, United States

Site Status RECRUITING

Indiana University

Bloomington, Indiana, United States

Site Status RECRUITING

Kansas University Medical Center

Kansas City, Kansas, United States

Site Status RECRUITING

Johns Hopkins University

Baltimore, Maryland, United States

Site Status RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status NOT_YET_RECRUITING

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

University of Michigan

Ann Arbor, Michigan, United States

Site Status RECRUITING

Washington University at St. Louis

St Louis, Missouri, United States

Site Status NOT_YET_RECRUITING

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status RECRUITING

Seattle Children's Hospital

Seattle, Washington, United States

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

United States

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Evelyn Bord, BS

Role: CONTACT

617-919-6154

Callie Bacon, MPH

Role: CONTACT

617-919-7696

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Kathryn Monroe

Role: primary

James Fowler

Role: backup

Allison Keck

Role: primary

Katie Poch

Role: primary

Kimmy Renner

Role: primary

Jennifer Gafford

Role: primary

Sherry Wooldridge

Role: primary

Rebecca Davis

Role: primary

Ashleigh Streby

Role: primary

Eric Hunter

Role: backup

Lisa Bendy

Role: primary

Mary Heskett

Role: backup

Megan Behrman

Role: primary

Lawrence Scott

Role: backup

Josephine Yodzis

Role: primary

Ruobin Wei

Role: primary

Margot Hardcastle

Role: backup

Sara Carvalho

Role: primary

Dawn Kruse

Role: primary

Jessica Carey

Role: backup

Stacy Postma

Role: primary

Irma Bauer

Role: backup

Adrienne Dericco

Role: primary

Keianna Brown

Role: primary

Gracia Bridges

Role: primary

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

IRB-P00050262

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Trikafta in Cystic Fibrosis Patients
NCT03506061 COMPLETED PHASE2