Cystic FibrosisPhase 3NCT02742519
A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
A Phase 3 study testing ivacaftor in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 6 sites in 3 countries.
Trial summary
To evaluate the efficacy of ivacaftor treatment, as measured by lung clearance index (LCI), in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance regulator (CFTR) gating mutation Read more about this study
- Who is it for?
- Ages 3 to 5 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- ivacaftor
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 14 participants
- Where
- 6 sitesin 3 countries
- Study dates
- May 2016to Aug 2017
- 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 3 to 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female with confirmed diagnosis of CF.
- Must have 1 of the following CFTR gating mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.
- Hematology, serum chemistry, and coagulation at Screening with no clinically significant abnormalities or concomitant diagnosis that would interfere with the LCI and CT scan study assessments, as judged by the investigator.
Who cannot join
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1
- Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject (in the opinion of the investigator)
- Abnormal liver function, at Screening, defined as ≥3 × upper limit of normal (ULN), of any 3 or more of the following: serum aspartate transaminase (AST), serum alanine transaminase (ALT), gamma-glutamyl transpeptidase (GGT), serum alkaline phosphatase (ALP), and total bilirubin
- History of solid organ or hematological transplantation
- Any clinically significant "non-CF-related" illness within 2 weeks before Day 1
- Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A within 2 weeks before Day 1
- Participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives (whichever is longer or as determined by the local requirements) before Screening
Trial locations and contacts
6 sites in 3 countries.
6 sites in 3 countries
Australia4 sites
Study site
Parkville, Victoria
Study site
South Brisbane
Study site
Subiaco
Study site
Westmead
About this study
Official title: A Phase 3b, 2-part, Randomized, Double-blind, Placebo-controlled Crossover Study With a Long-term Open-label Period to Investigate Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
To evaluate the efficacy of ivacaftor treatment, as measured by lung clearance index (LCI), in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance regulator (CFTR) gating mutation
- Conditions
- Cystic Fibrosis
- Being tested
- ivacaftor, 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.
ivacaftorDrug
Also called VX-770
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| Part 1-Sequence 1 | Experimental | ivacaftor in Treatment Period 1 →washout→placebo in Treatment Period 2 Receives: Drug: ivacaftor; Drug: Placebo |
| Part 1 - Sequence 2 | Experimental | placebo in Treatment Period 1→washout→ivacaftor in Treatment Period 2 Receives: Drug: ivacaftor; Drug: Placebo |
| Part 2: ivacaftor | Experimental | open label period Receives: Drug: ivacaftor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absolute Change From Baseline in Lung Clearance Index (LCI2.5) Through 8 Weeks of Treatment (Average of Week 4 and Week 8 LCI2.5)
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: Baseline Through Week 8 for each treatment period, Up to 24 Weeks
Secondary outcomes (5)
Absolute Change From Baseline in Immunoreactive Trypsinogen Levels at Week 8
Serum samples were collected for evaluation of change in immunoreactive trypsinogen levels at Week 8.
Measured: Baseline and Week 8 of each treatment period, Up to 24 Weeks
Absolute Change From Baseline in Fecal Elastase-1 Levels at Week 8
Fecal elastase-1 was used clinically to diagnose pancreatic exocrine insufficiency in participants with cystic fibrosis.
Measured: Baseline and Week 8 of each treatment period, Up to 24 Weeks
Absolute Change From Baseline in Weight at Week 8
Measured: Baseline and Week 8 of each treatment period, Up to 24 Weeks
Absolute Change From Baseline in Body Mass Index (BMI) at Week 8
BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2).
Measured: Baseline and Week 8 of each treatment period, Up to 24 Weeks
Number of Subjects With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Baseline up to Month 15
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2016 (estimated)
- Study startMay 2016
- Primary completionAug 2017
- Study completionAug 2017
Why it stopped: Low enrollment.
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
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 14 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
- NCT02742519
- Sponsor's protocol ID
- VX15-770-123
- Other IDs
- 2015-001267-39, 2015
- Status last verified
- Oct 2018
- First submitted
- 14 Apr 2016
- Last updated
- 19 Nov 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3b, 2-part, Randomized, Double-blind, Placebo-controlled Crossover Study With a Long-term Open-label Period to Investigate Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
- ClinicalTrials.gov ID
- NCT02742519
- Acronym / sponsor ID
- VX15-770-123
- Conditions
- Cystic Fibrosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Low enrollment.
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- May 2016
- Primary completion
- Aug 2017 (actual)
- Study completion
- Aug 2017 (actual)
- First posted
- 19 Apr 2016 (estimated)
- Results
- Posted 19 Nov 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate the efficacy of ivacaftor treatment, as measured by lung clearance index (LCI), in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance regulator (CFTR) gating mutation
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Part 1-Sequence 1 | Experimental | Drug: ivacaftor, Drug: Placeboivacaftor in Treatment Period 1 →washout→placebo in Treatment Period 2 |
| Part 1 - Sequence 2 | Experimental | Drug: ivacaftor, Drug: Placeboplacebo in Treatment Period 1→washout→ivacaftor in Treatment Period 2 |
| Part 2: ivacaftor | Experimental | Drug: ivacaftoropen label period |
Who may qualify
- Ages
- 3 to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Male or female with confirmed diagnosis of CF.
- Must have 1 of the following CFTR gating mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.
- Hematology, serum chemistry, and coagulation at Screening with no clinically significant abnormalities or concomitant diagnosis that would interfere with the LCI and CT scan study assessments, as judged by the investigator.
Key exclusion criteria (7 listed)
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1
- Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject (in the opinion of the investigator)
- Abnormal liver function, at Screening, defined as ≥3 × upper limit of normal (ULN), of any 3 or more of the following: serum aspartate transaminase (AST), serum alanine transaminase (ALT), gamma-glutamyl transpeptidase (GGT), serum alkaline phosphatase (ALP), and total bilirubin
- History of solid organ or hematological transplantation
- Any clinically significant "non-CF-related" illness within 2 weeks before Day 1
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 |
|---|---|---|
| ivacaftorAlso: VX-770 | Drug | Part 1 - Sequence 2, Part 1-Sequence 1, Part 2: ivacaftor |
| Placebo | Drug | Part 1 - Sequence 2, Part 1-Sequence 1 |
Primary outcome
Absolute Change From Baseline in Lung Clearance Index (LCI2.5) Through 8 Weeks of Treatment (Average of Week 4 and Week 8 LCI2.5)
Time frame: Baseline Through Week 8 for each treatment period, Up to 24 Weeks
LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
5 secondary outcomes
Absolute Change From Baseline in Immunoreactive Trypsinogen Levels at Week 8
Time frame: Baseline and Week 8 of each treatment period, Up to 24 Weeks
Absolute Change From Baseline in Fecal Elastase-1 Levels at Week 8
Time frame: Baseline and Week 8 of each treatment period, Up to 24 Weeks
Absolute Change From Baseline in Weight at Week 8
Time frame: Baseline and Week 8 of each treatment period, Up to 24 Weeks
Absolute Change From Baseline in Body Mass Index (BMI) at Week 8
Time frame: Baseline and Week 8 of each treatment period, Up to 24 Weeks
Number of Subjects With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Baseline up to Month 15
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Nov 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
6 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ivacaftor in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 6 sites in 3 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 3 inclusion and 7 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: stopped early, as of the sponsor's last update on 19 Nov 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Australia, Canada, United Kingdom. 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 19 Nov 2018). 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 From Baseline in Lung Clearance Index (LCI2.5) Through 8 Weeks of Treatment (Average of Week 4 and Week 8 LCI2.5) (time frame: Baseline Through Week 8 for each treatment period, Up to 24 Weeks). 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 19 Nov 2018.