Restarting triple modulator therapy with close safety monitoring

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

RETRIAL is a prospective, longitudinal, observational, multi-site effort enrolling people with cystic fibrosis age 6 and older who modified or stopped ETI because of new or worsening neuropsychiatric symptoms or liver injury and who plan to start VTD. Participants are followed over time with daily diaries, periodic surveys delivered to smart devices, and targeted mental health and liver monitoring to detect adverse events. The protocol includes sub-cohorts focused on mental health, liver, and neuro outcomes and requires a brief baseline assessment before restarting VTD. Data are collected across multiple U.S. CF centers to describe safety signals and patterns after restarting triple-modulator therapy.

Why it matters

Who can join: the full rules

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 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.

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:

Inclusion Criteria:

* 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:

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

Where it's running

• University of Alabama at Birmingham
  Birmingham, Alabama, United States Recruiting
• Children's Hospital Colorado
  Aurora, Colorado, United States Recruiting
• National Jewish Health
  Denver, Colorado, United States Recruiting
• Nemours Children's
  Wilmington, Delaware, United States Recruiting
• Nemours Children's
  Jacksonville, Florida, United States Recruiting
• Nemours Children's
  Orlando, Florida, United States Recruiting

Study contacts

• Principal investigator: Anna Georgiopoulos, MD — Massachusetts General Hospital
• Study coordinator: Evelyn Bord, BS
• Email: evelyn.bord@childrens.harvard.edu
• Phone: 617-919-6154

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.