Effects of elexacaftor-tezacaftor-ivacaftor on metabolism, epigenetics, and gut bacteria in people with cystic fibrosis
Impact of Elexacaftor-Tezacaftor-Ivacaftor Treatment on Metabolic, Epigenetic and Fecal Microbiota Profiles in People With Cystic Fibrosis.
This project will test whether starting elexacaftor-tezacaftor-ivacaftor (ETI) changes blood metabolism, epigenetic markers, and the gut microbiome in people with cystic fibrosis.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 150 (estimated) |
| Ages | 2 Years and up |
| Sex | All |
| Sponsor | Meyer Children's Hospital IRCCS Academic / other |
| Locations | 6 sites (Bari, Bari and 5 other locations) |
| Trial ID | NCT07363304 on ClinicalTrials.gov |
What this trial studies
This observational, multicenter Italian project will enroll people with cystic fibrosis who are about to begin ETI therapy and will collect biological samples before and after treatment initiation. Researchers will analyze blood for metabolic and epigenetic markers and stool samples to profile fecal microbiota composition. Serial sampling and laboratory analyses will be used to detect treatment-associated shifts in metabolism, DNA methylation or other epigenetic signals, and microbial community structure. The goal is to map systemic and gut-level changes that occur as patients start ETI under real-world clinic conditions.
Who should consider this trial
Good fit: Ideal candidates are people with cystic fibrosis of any age who are about to start ETI therapy under Italian marketing authorization and can provide informed consent and follow-up samples at a participating CF center.
Not a fit: Patients who are not starting ETI or who are already receiving ETI will not be eligible and thus are unlikely to receive any direct benefit from this project.
Why it matters
Potential benefit: If successful, this work could identify metabolic, epigenetic, or microbiome changes linked to ETI that help clinicians monitor patients, anticipate side effects, and tailor nutritional or microbial interventions.
How similar studies have performed: ETI has produced clear clinical benefits such as improved lung function and nutrition, and some recent reports note metabolic changes, but effects on epigenetic marks and the fecal microbiome remain incompletely characterized.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients with CF, of any age, about to start ETI therapy, in accordance with marketing authorization Italian legislative directives, followed at the participating CF centers. * Obtaining informed consent. Exclusion Criteria: * CF patients not in ETI therapy and CF patients already in ETI therapy.
Where this trial is running
Bari, Bari and 5 other locations
- Centro per la Fibrosi Cistica, Azienda Universitaria Ospedaliera Consorziale Policlinico — Bari, Bari, Italy (Not_yet_recruiting)
- Dipartimento di Pediatria, Centro Fibrosi Cistica, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico — Milan, Milano, Italy (Not_yet_recruiting)
- Unità Pediatrica, Dipartimento di Scienze Mediche Traslazionali, Centro di Riferimento Regionale per la Fibrosi Cistica, Università degli Studi di Napoli Federico II — Naples, Napoli, Italy (Not_yet_recruiting)
- Ospedale pediatrico Bambino Gesù, IRCCS, Dipartimento Pediatrico Universitario Ospedaliero, UOC Pneumologia e Fibrosi Cistica — Roma, Roma, Italy (Not_yet_recruiting)
- Centro per la Fibrosi Cistica, Ospedale Infantile Regina Margherita — Torino, Torino, Italy (Not_yet_recruiting)
- Azienda Ospedaliera Universitaria Meyer Istituto di Ricovero e Cura a Carattere Scientifico — Florence, Italy (Recruiting)
Study contacts
- Study coordinator: Vito Terlizzi, Medical Doctor
- Email: vito.terlizzi@meyer.it
- Phone: +39 055 566 2474
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.