A Phase 4 interventional study of Elexacaftor, Tezacaftor, Ivacaftor and Ivacaftor (VX-770) in Cystic Fibrosis (CF), sponsored by Istituto Giannina Gaslini. Recruiting at 1 site in Italy. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2026-10-14.
Sponsored by Istituto Giannina Gaslini · Phase 4, Interventional, and Other
Among 23 studies testing elexacaftor in Cystic Fibrosis — 2 open to participants now, 5 under way, 2 of them phase 3. elexacaftor · Cystic Fibrosis.
Among the 23 studies testing elexacaftor in Cystic Fibrosis, this one is the 22nd largest by planned enrollment, is the only one that started in 2026, is one of 3 in phase 4, and was the 23rd registered.
1,619 studies on the registry are indexed under Cystic Fibrosis; 199 are open to participants now.
This study's planned enrollment of 50 is above the median of 36 across 1,051 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →2 of the 23 studies testing elexacaftor in Cystic Fibrosis are open now, in 2 countries. Each count lists the studies.
elexacaftor: first FDA approval 2019 (TRIKAFTA (COPACKAGED)), first EMA approval 2020. What it is approved for, and how studies give it, is on its page →
Of the 154 Cystic Fibrosis studies recruiting now, 95 (62%) set no upper age limit and 43 (28%) stop at 75 or younger; 84 take children under 18; 41 take healthy volunteers.
This study: 6 Years and older · all sexes · no healthy volunteers.
The full table on the hub →23 studies; the count open now beside each. This study's row is marked.
First in 2017; this study’s year is marked. 1 started in 2026.
Median enrollment 120 across the 23 interventional studies. Of the 18 that completed or were terminated, 17 (94%) have results posted.
20 of 23 have an industry lead sponsor. The 4 most frequent:
Istituto Giannina Gaslini is the lead sponsor of 54 studies on the registry; 36 are open to participants now.
Istituto Giannina Gaslini has 2 studies filed under Cystic Fibrosis — fewer than ten, so there is no comparison to draw.
2 of the studies testing elexacaftor in Cystic Fibrosis that are under way reach primary completion within a year, 2 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05331183Phase 3Vertex Pharmaceuticals IncorporatedStudy to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del | 2027-04-06 |
| NCT06460506Phase 3Vertex Pharmaceuticals IncorporatedEvaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older | 2027-09-30 |
Among the 23 studies testing elexacaftor in Cystic Fibrosis, from their registry updates:
The goal of this clinical trial is to support the development of personalized therapy tailored to each patient's individual characteristics. A better understanding of the pharmacokinetic profiles of elexacaftor, tezacaftor, and ivacaftor is essential to support Therapeutic Drug Monitoring (TDM) and to guide dose adjustments when clinically indicated, without compromising therapeutic efficacy.
The study will include male and female patients aged ≥6 years with Cystic Fibrosis (CF) carrying at least one F508del mutation and receiving treatment with the elexacaftor/tezacaftor/ivacaftor combination for a sufficient period to achieve steady state.
The main objective of the study is to characterize the steady-state plasma concentration profiles of elexacaftor, tezacaftor, and ivacaftor in patients with Cystic Fibrosis treated with the elexacaftor/tezacaftor/ivacaftor combination.
Participants will continue their usual treatment with elexacaftor/tezacaftor/ivacaftor and ivacaftor according to the prescribed dosing schedule. During the study, they will attend regular clinical visits every 3 months for up to 12 months.
At these visits, blood samples will be collected as part of routine clinical care and used to measure the plasma concentrations of the study drugs. Additional blood samples may be collected after the morning dose to better characterize drug levels over time. In a subset of participants, additional blood samples may be collected at several time points during the day to further assess the pharmacokinetic profile of the drugs.
A single additional blood sample may be collected for pharmacogenetic analyses. Participants may also undergo a nasal brushing, as part of routine procedures, to obtain nasal epithelial cells for further analyses.
Clinical information, including routine laboratory tests, vital signs, treatment information and any adverse events, will be collected throughout the study. The results of the pharmacokinetic and other laboratory analyses will not be used to modify the participant's treatment, which will remain under the responsibility of the treating physician.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Elexacaftor, Tezacaftor, Ivacaftor · Drug: Ivacaftor (VX-770)
Drug pages: Elexacaftor · Tezacaftor · Ivacaftor
Cystic Fibrosis Transmembrane Regulator (CFTR) modulator therapy
Cystic Fibrosis Transmembrane Regulator (CFTR) modulator therapy
Plasma concentrations of elexacaftor, tezacaftor and ivacaftor at steady-state
Time frame: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]
Population pharmacokinetics (popPK) analysis of elexacaftor, tezacaftor and ivacaftor at steady state
Time frame: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]
Harvest of every Adverse Event (AE)/Adverse Drug Reaction (ADR)
Time frame: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]
Correlation between pharmacokinetics of elexacaftor, tezacaftor and ivacaftor and number of Adverse Event (AE)
Time frame: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]
Correlation between pharmacokinetics and elexacaftor, tezacaftor and ivacaftor efficacy in the 3 groups classifications (responders, non responders and unresolved responders) of elexacaftor, tezacaftor and ivacaftor
Time frame: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]
Started Sep 2026 (actual). Primary completion is estimated for Apr 2028, 19 months after the start.
Of 30 phase 4 interventional studies under Cystic Fibrosis, half reached primary completion within 32 months of starting (the median), counting those still running; a quarter within 20 and three quarters within 48. This study's planned 19 months is below that.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Studies of the drugs tested here: Elexacaftor · Tezacaftor · Ivacaftor
Istituto Giannina Gaslini