Cystic FibrosisPhase 3NCT03559062
A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis
A Phase 3 study testing TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 27 sites in 9 countries.
Trial summary
This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF). 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?
- 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
- 67 participants
- Where
- 27 sitesin 9 countries
- Study dates
- 17 May 2018to 21 Dec 2018
- 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
- Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
- Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
- Participants with a screening LCI2.5 result ≥7.5.
- Participants who are able to swallow tablets.
Who cannot join
- Clinically significant cirrhosis with or without portal hypertension.
- Colonization 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
27 sites in 9 countries.
27 sites in 9 countries
Germany8 sites
Universitaetsklinikum Koeln
Cologne
Universitaetsklinikum Essen
Essen
Klinikum der Johann Wolfgang Goethe-Universitaet
Frankfurt
Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen
Giessen
Medizinische Hochschule Hannover
Hanover
Universitaetsklinikum Heidelberg
Heidelberg
Universitaetsklinikum Jena
Jena
Universitaetsklinikum Tuebingen
Tübingen
United Kingdom5 sites
Royal Hospital for Sick Children
Edinburgh
Leeds General Infirmary
Leeds
Royal Brompton Hospital
London
Nottingham University Hospital City Campus
Nottingham
Southampton General Hospital
Southampton
Australia4 sites
Hunter Medical Research Institute (HMRI)
New Lambton Heights
Princess Margaret Hospital for Children
Perth
Lady Cilento Children's Hospital
South Brisbane
The Children's Hospital at Westmead
Westmead
Belgium2 sites
Universitair Ziekenhuis Brussel - Campus Jette
Brussels
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven
France2 sites
Groupe Hospitalier Pellegrin - Hôpital des Enfants
Bordeaux
Hôpital Necker - Enfants Malades
Paris
Ireland2 sites
Our Lady's Children's Hospital
Dublin
University Hospital Limerick
Limerick
Switzerland2 sites
Inselspital - Universitaetsspital Bern
Bern
Kinderspital Zuerich
Zurich
Poland1 site
Klinika Mukowiscydozy, Oddział Chorób Płuc SZP ZOZ
Dziekanów Leśny
About this study
Official title: A Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).
- Conditions
- Cystic Fibrosis
- Being tested
- TEZ/IVA, IVA, Placebo
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TEZ/IVADrug
Also called VX-661/VX-770, tezacaftor/ivacaftor fixed dose combination
Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg FDC tablet and those weighing ≥40 kg received TEZ 100 mg/IVA 150 mg FDC tablet.
IVADrug
Also called VX-770, ivacaftor
Participants weighing <40 kg IVA 75 mg tablet and those weighing ≥40 kg received IVA 150 mg tablet.
PlaceboDrug
Placebo matched to TEZ/IVA FDC
PlaceboDrug
Placebo matched to IVA
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Other | Participants with genotype F/F received placebo matched to TEZ/IVA fixed dose combination (FDC) in the morning and placebo matched to IVA in the evening for 8 weeks. Receives: Drug: Placebo |
| TEZ/IVA | Experimental | Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks. Receives: Drug: TEZ/IVA; Drug: IVA; Drug: Placebo |
| Ivacaftor | Experimental | Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks. Receives: Drug: IVA; Drug: Placebo |
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) Through Week 8
LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
Measured: From baseline through Week 8
Secondary outcomes (3)
Absolute Change in Sweat Chloride At Week 8
Sweat samples were collected using an approved collection device.
Measured: From baseline at Week 8
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Measured: From baseline through Week 8
Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit
Measured: From first dose of study drug up to safety follow-up visit (up to Week 12)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2018
- Study start17 May 2018
- Primary completion21 Dec 2018
- Study completion21 Dec 2018
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.
- Publications (2)Papers the sponsor has linked to this study.
- 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
- 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
- NCT03559062
- Sponsor's protocol ID
- VX16-661-115
- Other IDs
- 2016-004479-35, 2016
- Status last verified
- Feb 2020
- First submitted
- 5 Jun 2018
- Last updated
- 11 Feb 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
PubMed 37983082Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
PubMed 33331662
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
- ClinicalTrials.gov ID
- NCT03559062
- Acronym / sponsor ID
- VX16-661-115
- Conditions
- Cystic Fibrosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 67 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 17 May 2018 (actual)
- Primary completion
- 21 Dec 2018 (actual)
- Study completion
- 21 Dec 2018 (actual)
- First posted
- 15 Jun 2018 (actual)
- Results
- Posted 11 Feb 2020
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 of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo | Other | Drug: PlaceboParticipants with genotype F/F received placebo matched to TEZ/IVA fixed dose combination (FDC) in the morning and placebo matched to IVA in the evening for 8 weeks. |
| TEZ/IVA | Experimental | Drug: TEZ/IVA, Drug: IVA, Drug: PlaceboParticipants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks. |
| Ivacaftor | Experimental | Drug: IVA, Drug: PlaceboParticipants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks. |
Who may qualify
- Ages
- 6 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
- Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
- Participants with a screening LCI2.5 result ≥7.5.
- Participants who are able to swallow tablets.
Key exclusion criteria (4 listed)
- Clinically significant cirrhosis with or without portal hypertension.
- Colonization 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 |
|---|---|---|
| TEZ/IVAAlso: VX-661/VX-770, tezacaftor/ivacaftor fixed dose combinationParticipants weighing <40 kg received TEZ 50 mg/IVA 75 mg FDC tablet and those weighing ≥40 kg received TEZ 100 mg/IVA 150 mg FDC tablet. | Drug | TEZ/IVA |
| IVAAlso: VX-770, ivacaftorParticipants weighing <40 kg IVA 75 mg tablet and those weighing ≥40 kg received IVA 150 mg tablet. | Drug | Ivacaftor, TEZ/IVA |
| PlaceboPlacebo matched to TEZ/IVA FDC | Drug | Ivacaftor, Placebo |
| PlaceboPlacebo matched to IVA | Drug | Placebo, TEZ/IVA |
Primary outcome
Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8
Time frame: From baseline through Week 8
LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
3 secondary outcomes
Absolute Change in Sweat Chloride At Week 8
Time frame: From baseline at Week 8
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8
Time frame: From baseline through Week 8
Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit
Time frame: From first dose of study drug up to safety follow-up visit (up to Week 12)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Feb 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37983082Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
- Derived from this study: PubMed 33331662Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
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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.
27 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 27 sites in 9 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 4 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 11 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in Germany, United Kingdom, Australia, Belgium, France and 4 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. 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 11 Feb 2020). 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) Through Week 8 (time frame: From baseline through Week 8). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03559062, which also lists 2 linked publications.
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 11 Feb 2020.