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

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.

Description

Eligibility

Age range

6+ years

Sex

All

Healthy volunteers

No

Interventions

• Other
  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).

• 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.

• Other
  RETRIAL-Neuro

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

Locations (18)