Cystic FibrosisPhase 3NCT04353817
A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes
A Phase 3 study testing ELX/TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 34 sites in 10 countries.
Trial summary
This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes). Read more about this study
- Who is it for?
- Ages 6 to 11 years
- 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?
- Yes, some participants may receive a placebo
- How many people
- 121 participants
- Where
- 34 sitesin 10 countries
- Study dates
- 19 Jun 2020to 17 May 2021
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 6 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Heterozygous for the F508del mutation (F/MF)
- Forced expiratory volume in 1 second (FEV1) value greater than equal to(≥) 70%
Who cannot join
- Clinically significant cirrhosis with or without portal hypertension
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- Solid organ or hematological transplantation
- Other protocol defined Inclusion/Exclusion criteria may apply
Trial locations and contacts
34 sites in 10 countries.
34 sites in 10 countries
Germany7 sites
Charite Paediatric Pulmonology Department
Berlin
Universitaetsklinkum Koeln, CF-Studienzentrum
Cologne
Universitätsklinikum Essen
Essen
Johann Wolfgang Goethe University
Frankfurt
Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin
Giessen
Medizinische Hochschule Hannover
Hanover
Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
Heidelberg
United Kingdom7 sites
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
Bristol
Children's Hospital of Wales
Cardiff
Royal Hospital for Sick Children
Edinburgh
Alder Hey Children's NHS Foundation Trust
Liverpool
Great Ormond Street Hospital for Sick Children
London
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
London
Southampton General Hospital
Southampton
France5 sites
Groupe Hospitaler Pellegrin, CHU De Bordeaux
Bordeaux
CHU Lyon - Hopital Femme Mere-Enfant
Bron
Hopital Necker, Enfants Malades
Paris
Hopital Robert Debre
Paris
Centre de Perharidy
Roscoff
Australia3 sites
Telethon Kids Institute
Nedlands
Queensland Children's Hospital
South Brisbane
The Children's Hospital at Westmead
Westmead
Canada3 sites
McGill University Health Centre, Glen Site, Montreal Children's Hospital
Montreal
The Hospital for Sick Children
Toronto
British Columbia Children's Hospital
Vancouver
Israel2 sites
Hadassah University Hospital Mount Scopus
Jerusalem
Schneider Children's Medical Center
Petah Tikva
Netherlands2 sites
Universitair Medisch Centrum Groningen
Groningen
Erasmus Medical Center / Sophia Children's Hospital
Rotterdam
Spain2 sites
Hospital Universitari Vall d Hebron
Barcelona
Hospital Virgen de la Arrixaca
Murcia
Switzerland2 sites
Inselspital - Universitaetsspital Bern
Bern
Kinderspital Zuerich
Zurich
About this study
Official title: A Phase 3b, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes).
- Conditions
- Cystic Fibrosis
- Being tested
- ELX/TEZ/IVA, IVA, Placebo (matched to ELX/TEZ/IVA), Placebo (matched to 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 tablet for oral administration qd in the morning.
IVADrug
Also called VX-770, ivacaftor
Tablet for oral administration qd in the evening.
Placebo (matched to ELX/TEZ/IVA)Other
Placebo matched to ELX/TEZ/IVA for oral administration once daily (qd) in the morning.
Placebo (matched to IVA)Other
Placebo matched to IVA for oral administration qd in the evening.
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Participants received placebo matched to ELX/TEZ/IVA and placebo matched to IVA in the treatment period for 24 weeks. Receives: Other: Placebo (matched to ELX/TEZ/IVA); Other: Placebo (matched to IVA) |
| ELX/TEZ/IVA | Experimental | Participants weighing less than (<) 30 kilograms (kg) at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg every 12 hours (q12h) and participants weighing greater than equals to (>=) 30 kg at screening received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. Receives: Drug: ELX/TEZ/IVA; Drug: IVA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absolute Change in Lung Clearance Index 2.5 (LCI2.5)
The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
Measured: From Baseline Through Week 24
Secondary outcomes (2)
Absolute Change in Sweat Chloride (SwCl)
Sweat samples were collected using an approved collection device.
Measured: From Baseline Through Week 24
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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2020
- Study start19 Jun 2020
- Primary completion17 May 2021
- Study completion17 May 2021
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 121 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
- NCT04353817
- Sponsor's protocol ID
- VX19-445-116
- Other IDs
- 2019-003554-86, 2019
- Status last verified
- Jul 2022
- First submitted
- 16 Apr 2020
- Last updated
- 26 Jul 2022
- 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
Study at a glance
From the registry record, last updated by the sponsor on 26 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3b, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
- ClinicalTrials.gov ID
- NCT04353817
- Acronym / sponsor ID
- VX19-445-116
- Conditions
- Cystic Fibrosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 121 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 19 Jun 2020 (actual)
- Primary completion
- 17 May 2021 (actual)
- Study completion
- 17 May 2021 (actual)
- First posted
- 20 Apr 2020 (actual)
- Results
- Posted 26 Jul 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Other: Placebo (matched to ELX/TEZ/IVA), Other: Placebo (matched to IVA)Participants received placebo matched to ELX/TEZ/IVA and placebo matched to IVA in the treatment period for 24 weeks. |
| ELX/TEZ/IVA | Experimental | Drug: ELX/TEZ/IVA, Drug: IVAParticipants weighing less than (<) 30 kilograms (kg) at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg every 12 hours (q12h) and participants weighing greater than equals to (>=) 30 kg at screening received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
Who may qualify
- Ages
- 6 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Heterozygous for the F508del mutation (F/MF)
- Forced expiratory volume in 1 second (FEV1) value greater than equal to(≥) 70%
Key exclusion criteria (4 listed)
- Clinically significant cirrhosis with or without portal hypertension
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- Solid organ or hematological transplantation
- Other protocol defined Inclusion/Exclusion criteria may apply
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ELX/TEZ/IVAAlso: VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftorFixed-dose combination tablet for oral administration qd in the morning. | Drug | ELX/TEZ/IVA |
| IVAAlso: VX-770, ivacaftorTablet for oral administration qd in the evening. | Drug | ELX/TEZ/IVA |
| Placebo (matched to ELX/TEZ/IVA)Placebo matched to ELX/TEZ/IVA for oral administration once daily (qd) in the morning. | Other | Placebo |
| Placebo (matched to IVA)Placebo matched to IVA for oral administration qd in the evening. | Other | Placebo |
Primary outcome
Absolute Change in Lung Clearance Index 2.5 (LCI2.5)
Time frame: From Baseline Through Week 24
The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
2 secondary outcomes
Absolute Change in Sweat Chloride (SwCl)
Time frame: From Baseline Through Week 24
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 28
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jul 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: 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
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
34 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ELX/TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 34 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 34 sites in Germany, United Kingdom, France, Australia, Canada and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Jul 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Absolute Change in Lung Clearance Index 2.5 (LCI2.5) (time frame: From Baseline Through Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jul 2022.