Cystic FibrosisPhase 4NCT01937325
CPET in CF Patients With One G551D Mutation Taking VX770
A Phase 4 study testing ivacaftor in people with Cystic Fibrosis, run by The Alfred at 1 site in Australia.
Trial summary
Ivacaftor will restore CFTR function in treated CF patients with the G551D mutation. Improvement in ventilation, salt balance and well-being will contribute to better exercise capacity at all levels of lung function. Read more about this study
- Who is it for?
- Ages 16 to 70 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- ivacaftor
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteAustralia
- Study dates
- Feb 2014to Dec 2016
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 16 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- All participants will have CF proven based on established criteria (sweat test, genotype and phenotype).
- All participants will have at least one copy of the G551D mutation.
- All will be able to perform an exercise study and complete study questionnaires and assessments.
- Age range will be between 16 and 75 years of age.
- Lung function inclusion will be above 25% predicted FEV1.
Who cannot join
- Participants will not be included if they are unable to complete study assessments or have had a known adverse reaction to Ivacaftor.
- Female participants will be excluded if found to return a positive pregnancy test at screening.
- Participants will be excluded if using St. John's Wort or rifampicin (strong CYP3A inducers).
- Participants with significant liver dysfunction will be excluded (ALT or ALT above 5 times upper limit of normal).
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Airway Infection, Inflammatory Markers and Exercise Capacity in Patients With Cystic Fibrosis and at Least One G551D Mutation Taking VX770 (Ivacaftor)
Ivacaftor will restore CFTR function in treated CF patients with the G551D mutation. Improvement in ventilation, salt balance and well-being will contribute to better exercise capacity at all levels of lung function. While potential improvements may be variable across the spectrum of lung function, even small gains at low levels of FEV1 may have significant benefit for some subjects.
Read the detailed description
The Alfred CF Service is ready to initiate therapy with VX-770 in it's cohort. Investigators therefore have an opportunity to examine correlates between improvements in lung function, exercise capacity, inflammatory markers and indices of wellbeing in CF that may not be repeatable in a single cohort.
Investigators propose a double-blind, placebo-controlled cross-over study of 20 patients currently awaiting initiation of ivacaftor therapy. Patients enrolled in the study will be asked to undergo screening (day -28), and baseline assessments (day 0) and re-assessment studies (day 28) after treatment period 1, followed by baseline assessment at the beginning of treatment period 2 (day 56) and at the conclusion of treatment period 2 (day 84). A further assessment (day 224) will be performed 140 (+/-7) days following commencement on open label Ivacaftor.
After satisfying eligibility criteria, subjects will be randomly assigned to initial active treatment or placebo following a 4 week run-in period. After completion of period 1 and a 4 week washout period, subjects will cross-over to the alternative treatment. After 4 weeks of period 2, subjects will undergo final assessment as shown in the diagram below. However participants choosing not to continue taking ivacaftor in a Named Patient Program or another similar program run by Vertex Pharmaceuticals, Inc., ('Vertex'), at the end of the study period will be required to undergo a Safety Follow-Up visit 28 days after the final dose of study drug. Safety Follow-Up assessments will not include the CPET.
- Conditions
- Cystic Fibrosis
- Being tested
- ivacaftor, placebo
- Keywords
- Eligible CF patients with G551D gene mutation
- 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 Kalydeco
active arm
placeboDrug
Also called ivacaftor matched placebo
active arm
| Study group | Role | What they receive |
|---|---|---|
| Ivacaftor | Active comparator | 150mg orally twice daily Receives: Drug: ivacaftor |
| Placebo | Placebo comparator | Matching placebo Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
exercise capacity
Respiratory exercise testing, including spirometry and V02 max.
Measured: one month, 3 months
Secondary outcomes (1)
Inflammatory profile
Cytokine levels (IL-1β, IL-6, TNFα, IL-8, VEGF \& Activin A) determined using cytometric bead analysis and / or ELISA
Measured: One month, 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2013 (estimated)
- Study startFeb 2014
- Primary completionDec 2014
- Study completionDec 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Investigator, Outcomes assessor
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Alfred
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, John Wilson, The Alfred
- Investigators
- John Wilson, Principal investigator, Alfred Health and Monash University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01937325
- Sponsor's protocol ID
- CPET in CF
- Status last verified
- Jul 2016
- First submitted
- 4 Sep 2013
- Last updated
- 25 Jul 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Edgeworth D, Keating D, Ellis M, Button B, Williams E, Clark D, Tierney A, Heritier S, Kotsimbos T, Wilson J. Improvement in exercise duration, lung function and well-being in G551D-cystic fibrosis patients: a double-blind, placebo-controlled, randomized, cross-over study with ivacaftor treatment. Clin Sci (Lond). 2017 Jul 16;131(15):2037-2045. doi: 10.1042/CS20170995. Print 2017 Aug 1.
PubMed 28611235
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Airway Infection, Inflammatory Markers and Exercise Capacity in Patients With Cystic Fibrosis and at Least One G551D Mutation Taking VX770 (Ivacaftor)
- ClinicalTrials.gov ID
- NCT01937325
- Acronym / sponsor ID
- CPET in CF
- Conditions
- Cystic Fibrosis
- Keywords
- Eligible CF patients with G551D gene mutation
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (actual)
- Lead sponsor
- The AlfredUniversity, hospital or other
- Study start
- Feb 2014
- Primary completion
- Dec 2014 (actual)
- Study completion
- Dec 2016 (estimated)
- First posted
- 9 Sep 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Ivacaftor will restore CFTR function in treated CF patients with the G551D mutation. Improvement in ventilation, salt balance and well-being will contribute to better exercise capacity at all levels of lung function. While potential improvements may be variable across the spectrum of lung function, even small gains at low levels of FEV1 may have significant benefit for some subjects.
Detailed description
The Alfred CF Service is ready to initiate therapy with VX-770 in it's cohort. Investigators therefore have an opportunity to examine correlates between improvements in lung function, exercise capacity, inflammatory markers and indices of wellbeing in CF that may not be repeatable in a single cohort.
Investigators propose a double-blind, placebo-controlled cross-over study of 20 patients currently awaiting initiation of ivacaftor therapy. Patients enrolled in the study will be asked to undergo screening (day -28), and baseline assessments (day 0) and re-assessment studies (day 28) after treatment period 1, followed by baseline assessment at the beginning of treatment period 2 (day 56) and at the conclusion of treatment period 2 (day 84). A further assessment (day 224) will be performed 140 (+/-7) days following commencement on open label Ivacaftor.
After satisfying eligibility criteria, subjects will be randomly assigned to initial active treatment or placebo following a 4 week run-in period. After completion of period 1 and a 4 week washout period, subjects will cross-over to the alternative treatment. After 4 weeks of period 2, subjects will undergo final assessment as shown in the diagram below. However participants choosing not to continue taking ivacaftor in a Named Patient Program or another similar program run by Vertex Pharmaceuticals, Inc., ('Vertex'), at the end of the study period will be required to undergo a Safety Follow-Up visit 28 days after the final dose of study drug. Safety Follow-Up assessments will not include the CPET.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ivacaftor | Active comparator | Drug: ivacaftor150mg orally twice daily |
| Placebo | Placebo comparator | Drug: placeboMatching placebo |
Who may qualify
- Ages
- 16 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- All participants will have CF proven based on established criteria (sweat test, genotype and phenotype).
- All participants will have at least one copy of the G551D mutation.
- All will be able to perform an exercise study and complete study questionnaires and assessments.
- Age range will be between 16 and 75 years of age.
- Lung function inclusion will be above 25% predicted FEV1.
Key exclusion criteria (4 listed)
- Participants will not be included if they are unable to complete study assessments or have had a known adverse reaction to Ivacaftor.
- Female participants will be excluded if found to return a positive pregnancy test at screening.
- Participants will be excluded if using St. John's Wort or rifampicin (strong CYP3A inducers).
- Participants with significant liver dysfunction will be excluded (ALT or ALT above 5 times upper limit of normal).
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: Kalydecoactive arm | Drug | Ivacaftor |
| placeboAlso: ivacaftor matched placeboactive arm | Drug | Placebo |
Primary outcome
exercise capacity
Time frame: one month, 3 months
Respiratory exercise testing, including spirometry and V02 max.
1 secondary outcome
Inflammatory profile
Time frame: One month, 3 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2016.
- Derived from this study: PubMed 28611235Edgeworth D, Keating D, Ellis M, Button B, Williams E, Clark D, Tierney A, Heritier S, Kotsimbos T, Wilson J. Improvement in exercise duration, lung function and well-being in G551D-cystic fibrosis patients: a double-blind, placebo-controlled, randomized, cross-over study with ivacaftor treatment. Clin Sci (Lond). 2017 Jul 16;131(15):2037-2045. doi: 10.1042/CS20170995. Print 2017 Aug 1.
- Individual participant dataSharing planned: No
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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.
John Wilson principal investigator
Alfred Health and Monash University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing ivacaftor in people with Cystic Fibrosis, run by The Alfred at 1 site in Australia.
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 5 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: status not confirmed, as of the sponsor's last update on 25 Jul 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
exercise capacity (time frame: one month, 3 months). 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 NCT01937325, which also lists 1 linked publication.
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 25 Jul 2016; AzTrials last refreshed its copy on 5 Nov 2026.