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

Study at a glance

From the registry record, last updated by the sponsor on 3 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title

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

ClinicalTrials.gov ID

NCT06683092

Acronym / sponsor ID

IRB-P00050262

Conditions

Cystic Fibrosis

Keywords

patient care, adverse events, monitoring

Study type

Observational

Recruitment status

Recruiting

Enrolment

200 participants (estimated)

Lead sponsor

Boston Children's HospitalUniversity, hospital or other

Collaborators

Cystic Fibrosis Foundation, Johns Hopkins University, Massachusetts General Hospital, National Jewish Health, Indiana University, Children's Hospital Colorado, University of Kansas Medical Center, Washington University School of Medicine

Study start

1 May 2025 (actual)

Primary completion

Mar 2028 (estimated)

Study completion

Jun 2028 (estimated)

First posted

12 Nov 2024 (actual)

Results

Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Observational model

Cohort

Time perspective

Other

Number of arms

3

Who may qualify

Ages

6 years and older

Sex

All

Healthy volunteers

Not accepted

Age groups

Child, Adult, Older adult

Study population

Female and male people with CF ages 6 and older who are not able to tolerate the standard dose of ETI due to either new/worsening mental health symptoms and/or drug-induced liver injury.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Primary outcome

1. 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

   Discontinuation or change from VTD standard dosing due to mental health symptoms at any time during the one-year follow-up as reported in either: daily diaries, biweekly or quarterly surveys, or chart review.

2. RETRIAL-Mental Health (2nd objective - symptoms): proportion of participants that experience any worsening in mental health symptoms during the 6-month follow-up

   Time frame: Baseline to Month 6

   Change from baseline that exceeds the minimally-important difference (MID) / minimally-important change (MIC) within the first 6 months after starting VTD in any of the following patient-reported outcome measures: (1) depression (PHQ-8 or PROMIS depression parent proxy), (2) anxiety (GAD-7 or PROMIS anxiety parent proxy), (3) anger and irritability (PROMIS-anger or PROMIS-anger parent proxy), (4) cognitive functioning (PROMIS- cognitive function or PROMIS-cognitive function parent proxy), (5) sleep disturbance (PROMIS-sleep disturbance or PROMIS-sleep disturbance parent proxy).

3. RETRIAL-Mental Health (3rd objective - RETRIAL-Neuro): proportion of participants that have a worsening on the NIH Toolbox's Fluid Cognition Composite score at 1 month follow-up.

   Time frame: Baseline to Day 30

   Decrease in age-adjusted standard score from baseline to 1 month on the NIH Toolbox's Fluid Cognition Composite Score (a performance-based neurocognitive measure) that exceeds MIC ≥ 7.5 points, signifying a worsening in function.

4. RETRIAL-Liver: one-year incidence of drug-induced liver injury (DILI) with VTD, defined by expert consensus after review of clinical and laboratory data.

   Time frame: Baseline to Month 12

   Positive DILI diagnosis, including those characterized as "likely," "probable," and "highly probable," as reviewed by a hepatology panel of three experts who assign these likelihood scores based upon review of case report forms, including a specific clinical narrative and laboratory values assessing for other potential etiologies.

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

• Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2025.
• Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.

18 sites in 1 country: see every site and its status.