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

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Boston Children's Hospital
Study ID
NCT06683092
Status
Recruiting
Apply to this trial

Conditions

  • Cystic Fibrosis

Eligibility Criteria

Sex
ALL
Age
6 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • RETRIAL-Mental Health — 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-Liver — 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.
  • RETRIAL-Neuro — OTHER
    Participants will complete a neurocognitive assessment prior to starting VTD and 28 days after.

Study Details

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.

Key Dates

Start date
May 1, 2025
Status verified
Oct 2025
Primary completion
Mar 31, 2028
Completion
Jun 30, 2028

Study Design

Enrollment
200 participants (estimated)

Arms

  • Arm: 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).
  • Arm: 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).
  • Arm: 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).

Primary Outcome Measure

RETRIAL-Mental Health (1st objective - discontinuation): one-year incidence of discontinuation or change from VTD standard dosing due to new/worsening mental health symptoms. [ Time Frame: Baseline to Month 12 ]

Central Contacts

Locations (18)

FacilityCityStateZIPSite coordinators
University of Alabama at BirminghamBirminghamAlabama35233
Kathryn Monroe
[email protected]
James Fowler
Children's Hospital ColoradoAuroraColorado80045
Allison Keck
[email protected]
Emma Lyons, PhD (PRINCIPAL_INVESTIGATOR)
National Jewish HealthDenverColorado80206
CJ Bathgate, PhD (PRINCIPAL_INVESTIGATOR)
Nemours Children'sWilmingtonDelaware19803
Kimmy Renner
[email protected]
Kimberly Canter, PhD (PRINCIPAL_INVESTIGATOR)
David Fedele, PhD (PRINCIPAL_INVESTIGATOR)
Nemours Children'sJacksonvilleFlorida32207
Jennifer Gafford
[email protected]
Kimberly Canter, PhD (PRINCIPAL_INVESTIGATOR)
David Fedele, PhD (PRINCIPAL_INVESTIGATOR)
Nemours Children'sOrlandoFlorida32827
Sherry Wooldridge
[email protected]
Kimberly Canter, PhD (PRINCIPAL_INVESTIGATOR)
David Fedele, PhD (PRINCIPAL_INVESTIGATOR)
Nemours Children'sPensacolaFlorida32514
Rebecca Davis
[email protected]
Kimberly Canter, PhD (PRINCIPAL_INVESTIGATOR)
David Fedele, PhD (PRINCIPAL_INVESTIGATOR)
Emory UniversityAtlantaGeorgia30322
Ashleigh Streby
[email protected]
Eric Hunter
Kimberly Dickinson, MD (PRINCIPAL_INVESTIGATOR)
Indiana UniversityBloomingtonIndiana47405
Mary Heskett
Emma Tillman, PhD PharmD (PRINCIPAL_INVESTIGATOR)
Kansas University Medical CenterKansas CityKansas66160
Megan Behrman
[email protected]
Lawrence Scott
[email protected]
Amanda Bruce, PhD (PRINCIPAL_INVESTIGATOR)
Johns Hopkins UniversityBaltimoreMaryland21287
Josephine Yodzis
[email protected]
Noah Lechtzin, MD (PRINCIPAL_INVESTIGATOR)
Boston Children's HospitalBostonMassachusetts02215
Sara Carvalho
[email protected]
Carolyn Snell, PhD (PRINCIPAL_INVESTIGATOR)
Mimi Stotsky, PhD (PRINCIPAL_INVESTIGATOR)
Massachusetts General HospitalBostonMassachusetts02114
Ruobin Wei
Margot Hardcastle
[email protected]
Isabel Neuringer, MD (PRINCIPAL_INVESTIGATOR)
University of MichiganAnn ArborMichigan48109
Jessica Carey
Hanna Phan, PharmD (PRINCIPAL_INVESTIGATOR)
Washington University at St. LouisSt LouisMissouri63130
Stacy Postma
Irma Bauer
[email protected]
Andrea Coverston, MD (PRINCIPAL_INVESTIGATOR)
University of Pittsburgh Medical CenterPittsburghPennsylvania15224
Adrienne Dericco
[email protected]
Traci Kazmerski, MD (PRINCIPAL_INVESTIGATOR)
University of Texas Southwestern Medical CenterDallasTexas75235
Keianna Brown
[email protected]
David Finklea, MD (PRINCIPAL_INVESTIGATOR)
Meghana Sathe, MD (PRINCIPAL_INVESTIGATOR)
Seattle Children's HospitalSeattleWashington98105
Gracia Bridges
[email protected]
Freda Liu, PhD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Birmingham, AL

By research site
University of Alabama at Birmingham· Birmingham, ALChildren's Hospital Colorado· Aurora, CONational Jewish Health· Denver, CONemours Children's· Wilmington, DENemours Children's· Jacksonville, FLNemours Children's· Orlando, FL

Related Studies