Cystic FibrosisPhase 3NCT05882357
Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age
A Phase 3 study testing ELX/TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 19 sites in 7 countries.
Trial summary
The study evaluates the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF participants 2 to less than (<) 24 months of age. Read more about this study
- Who is it for?
- Ages 12 to 24 months
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- ELX/TEZ/IVA and IVA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 67 participants
- Where
- 19 sitesin 7 countries
- Study dates
- 27 Jun 2023to 4 Sep 2025
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 24 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participants who have at least 1 F508del mutation in the CF transmembrane conductance regulator (CFTR) gene or another ELX/TEZ/IVA-responsive CFTR mutation
Who cannot join
- History of any illness or any clinical condition that, in the opinion of the investigator, might either confound the results of the study or pose an additional risk in administering study drug(s) to the participant
- Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
19 sites in 7 countries.
19 sites in 7 countries
United Kingdom6 sites
Children and Young Adults Research Unit
Cardiff
Leeds General Infirmary
Leeds
Alder Hey Children's NHS Foundation Trust
Liverpool
Great Ormond Street Hospital for Children
London
Royal Brompton Hospital
London
Southampton General Hospital
Southampton
Australia4 sites
Telethon Kids Institute
Nedlands
The Royal Children's Hospital
Parkville
Queensland Children's Hospital
South Brisbane
The Children's Hospital at Westmead
Westmead
Germany3 sites
Charite Paediatric Pulmonology Department
Berlin
Kinderklinik III, Abt. fur Pneumologie
Essen
Medizinische Hochschule Hannover
Hanover
Canada2 sites
The Hospital for Sick Children
Toronto
British Columbia Children's Hospital
Vancouver
Switzerland2 sites
Inselspital - Universitaetsspital Bern
Bern
Kinderspital Zuerich
Zurich
Netherlands1 site
Erasmus Medical Center / Sophia Children's Hospital
Rotterdam
About this study
Official title: A Phase 3, Open-label Study Evaluating the Pharmacokinetics, Safety, and Tolerability of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 12 to Less Than 24 Months of Age
The study evaluates the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF participants 2 to less than (<) 24 months of age.
- Conditions
- Cystic Fibrosis
- Being tested
- ELX/TEZ/IVA, IVA
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ELX/TEZ/IVADrug
Also called VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor
Fixed-dose combination granules for oral administration.
IVADrug
Also called VX-770, ivacaftor
Granules for oral administration
| Study group | Role | What they receive |
|---|---|---|
| Part A: ELX/TEZ/IVA | Experimental | Participants received ELX 40 milligram (mg)/ TEZ 20 mg/IVA 30 mg fixed-dose combination (FDC) granules once every morning (qAM) and 25 mg IVA once every evening (qPM) for 15 Days. Receives: Drug: ELX/TEZ/IVA; Drug: IVA |
| Part B: ELX/TEZ/IVA | Experimental | Participants received one of three dosing regimens based on their body weight at Day 1 . Dosage 1: ELX 80 mg qAM/TEZ 40 mg qAM /IVA 60 mg qAM FDC granules and 59.5 IVA mg qPM; Dosage 2: ELX 60 mg qAM /TEZ 30 mg qAM /IVA 45 mg qAM FDC granules and 50 mg IVA qPM; Dosage 3: ELX 40 mg qAM / TEZ 20 mg qAM /IVA 30 mg qAM FDC granules and 25mg IVA qPM. Part B included some participants from Part A and newly enrolled participants. Receives: Drug: ELX/TEZ/IVA; Drug: IVA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and Their Relevant Metabolites (M23-ELX, M1-TEX, M1-IVA)
Measured: At Day 15
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Measured: Day 1 up to Day 43
Part B : Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Measured: Day 1 up to Week 28
Secondary outcomes (2)
Part B: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and Their Relevant Metabolites (M23-ELX, M1-TEZ, M1-IVA)
Measured: Day 15, Week 4, 12 and 16
Part B: Absolute Change in Sweat Chloride (SwCl) From Baseline Through Week 24
Measured: From Baseline through Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 May 2023
- Study start27 Jun 2023
- Primary completion4 Sep 2025
- Study completion4 Sep 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 67 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05882357
- Sponsor's protocol ID
- VX22-445-122
- Other IDs
- 2023-503230-49-00, 2023
- Status last verified
- Oct 2026
- First submitted
- 19 May 2023
- Last updated
- 15 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing