Cystic FibrosisPhase 2NCT00865904
Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation
A Phase 2 study testing VX-809 in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 25 sites in 5 countries.
Trial summary
The primary objective of the study was to evaluate the safety and tolerability of VX-809 in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- VX-809
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 93 participants
- Where
- 25 sitesin 5 countries
- Study dates
- Mar 2009to Dec 2009
- Phase
- Phase 2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed diagnosis of CF with ∆F508-CFTR mutation in both alleles
- Forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent (%) of predicted normal for age, gender, and height
- Weight >=40 kilograms (kg) and body mass index greater than or equal to 18.5 kilogram per square meter (kg/m^2)
- Screening laboratory values, tests, and physical examination within acceptable ranges
- Negative pregnancy test (for women of child-bearing potential)
- Able and willing to follow contraceptive requirements
- Willing to remain on a stable medication regimen for the duration of study participation
Who cannot join
- History of any illness, or any ongoing acute illness, that could impact the safety of the study participant or may confound results of study
- Pulmonary exacerbation or changes in therapy for pulmonary disease within 14 days before receiving the first dose of study drug
- Impaired hepatic or renal function
- History of organ or hematological transplant
Trial locations and contacts
25 sites in 5 countries.
25 sites in 5 countries
United States18 sites
Study site
Atlanta, Georgia
Study site
Aurora, Colorado
Study site
Baltimore, Maryland
Study site
Birmingham, Alabama
Study site
Boston, Massachusetts
Study site
Chapel Hill, North Carolina
Study site
Chicago, Illinois
Study site
Cincinnati, Ohio
Study site
Cleveland, Ohio
Study site
Columbus, Ohio
Study site
Iowa City, Iowa
Study site
Minneapolis, Minnesota
Study site
Palo Alto, California
Study site
Philadelphia, Pennsylvania
Study site
Pittsburgh, Pennsylvania
Study site
San Diego, California
Study site
Seattle, Washington
Study site
St Louis, Missouri
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study of VX-809 to Evaluate Safety, Pharmacokinetics, and Pharmacodynamics of VX-809 in Cystic Fibrosis Subjects Homozygous for the ∆F508-CFTR Gene Mutation
The primary objective of the study was to evaluate the safety and tolerability of VX-809 in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
Read the detailed description
This was a Phase 2, randomized, double-blind, placebo-controlled, multiple-dose study of orally-administered VX-809 in participants with CF who are homozygous for the specific CFTR mutation known as ∆F508 or F508del. Enrollment was planned for 90 participants at approximately 20 centers. Participants were planned to be randomized in a 4:1 ratio to receive 1 of 4 doses of VX-809 or placebo once a day for 28 days in a parallel design. Participants were outpatients during the study, except for overnight stays on Day 1 and 28.
- Conditions
- Cystic Fibrosis
- Being tested
- VX-809, Placebo
- Keywords
- ∆F508, F508del, Cystic Fibrosis Transmembrane Conductance Regulator, CFTR, Pancreatic diseases, Lung diseases, Genetic disease, inborn, Infant, newborn, diseases
- Medical subject headings
- Cystic Fibrosis, Pancreatic Diseases, Lung Diseases, Genetic Diseases, Inborn, Infant, Newborn, Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-809Drug
Capsules
PlaceboDrug
Placebo matched to VX-809 capsules.
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo matched to VX-809 capsule orally once daily for 28 days. Receives: Drug: Placebo |
| VX-809, 25 mg | Experimental | VX-809, 25 milligram (mg) capsule orally once daily for 28 days. Receives: Drug: VX-809 |
| VX-809, 50 mg | Experimental | VX-809, 50 mg capsule orally once daily for 28 days. Receives: Drug: VX-809 |
| VX-809, 100 mg | Experimental | VX-809, 100 mg capsule orally once daily for 28 days. Receives: Drug: VX-809 |
| VX-809, 200 mg | Experimental | VX-809, 200 mg capsule orally once daily for 28 days. Receives: Drug: VX-809 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability Based on Adverse Events (AEs)
AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. Serious adverse event (SAE) (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Number of participants with AEs and SAEs are reported. An AE that started at or after initial dosing of study drug, or increased in severity after initial dosing of study drug visit is considered treatment-emergent.
Measured: Up to 14 days after last dose (last dose = Day 28)
Secondary outcomes (9)
Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Day 28
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Measured: Baseline, Day 28
Change From Baseline in Percent Predicted FEV1 at Day 28
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method.
Measured: Baseline, Day 28
Change From Baseline in Forced Vital Capacity (FVC) at Day 28
FVC is the volume of air that can be forcibly exhaled from the lungs after taking the deepest breath possible.
Measured: Baseline, Day 28
Change From Baseline in Forced Expiratory Flow Over the Middle Half of the FVC (FEF25-75) at Day 28
FEF25-75 is total volume of air exhaled from the lungs over the middle half of the FVC test, expressed as liters per second (L/sec).
Measured: Baseline, Day 28
Change From Baseline in Sweat Chloride at Day 28
Sweat samples were collected using an approved Macroduct (Wescor) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride.
Measured: Baseline, Day 28
Change From Baseline in Nasal Potential Difference (NPD) of Zero Chloride Plus Isoproterenol Response at Day 28
Nasal potential difference (NPD) provides a direct and sensitive evaluation of sodium and chloride transport in secretory epithelial cells via assessment of transepithelial bioelectric properties. NPD under conditions of zero chloride concentration perfusion solution in the presence of isoproterenol is reported.
NPDs were performed according to Cystic Fibrosis Foundation Therapeutics Development Network (CFFT TDN) Standard Operating Procedure (SOP) 528.00 "Standardization of Measurement of Nasal Membrane Transepithelial Potential Difference (NPD) - electronic data capture (EDC) and Perfusion or Perfusion-Free Probe".
Measured: Baseline, Day 28
Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Domain Scores at Day 28
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. CFQ-R domains include: Body, Digestion, Eat, Emotion, Health Perceptions, Physical, Respiratory, Role, Social, Treatment Burden, Vitality, and Weight. Individual domain score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Measured: Baseline, Day 28
Maximum Plasma Concentration (Cmax) of VX-809
Only participants who received VX-809 were analyzed for this outcome measure.
Measured: Day 1 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post-dose), Day 28 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, 24, and 30-60 hours post dose)
Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24) of VX-809
Only participants who received VX-809 were analyzed for this outcome measure.
Measured: Day 1 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post-dose), Day 28 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post dose)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2009 (estimated)
- Study startMar 2009
- Primary completionDec 2009
- Study completionDec 2009
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 (3)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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 93 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Vertex Pharmaceuticals Incorporated
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00865904
- Sponsor's protocol ID
- VX08-809-101
- Status last verified
- Jun 2015
- First submitted
- 18 Mar 2009
- Last updated
- 28 Aug 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
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 33331662Clancy JP, Rowe SM, Accurso FJ, Aitken ML, Amin RS, Ashlock MA, Ballmann M, Boyle MP, Bronsveld I, Campbell PW, De Boeck K, Donaldson SH, Dorkin HL, Dunitz JM, Durie PR, Jain M, Leonard A, McCoy KS, Moss RB, Pilewski JM, Rosenbluth DB, Rubenstein RC, Schechter MS, Botfield M, Ordonez CL, Spencer-Green GT, Vernillet L, Wisseh S, Yen K, Konstan MW. Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation. Thorax. 2012 Jan;67(1):12-8. doi: 10.1136/thoraxjnl-2011-200393. Epub 2011 Aug 8.
PubMed 21825083
Study at a glance
From the registry record, last updated by the sponsor on 28 Aug 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study of VX-809 to Evaluate Safety, Pharmacokinetics, and Pharmacodynamics of VX-809 in Cystic Fibrosis Subjects Homozygous for the ∆F508-CFTR Gene Mutation
- ClinicalTrials.gov ID
- NCT00865904
- Acronym / sponsor ID
- VX08-809-101
- Conditions
- Cystic Fibrosis
- Keywords
- ∆F508, F508del, Cystic Fibrosis Transmembrane Conductance Regulator, CFTR, Pancreatic diseases, Lung diseases, Genetic disease, inborn, Infant, newborn, diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 93 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- Mar 2009
- Primary completion
- Dec 2009 (actual)
- Study completion
- Dec 2009 (actual)
- First posted
- 19 Mar 2009 (estimated)
- Results
- Posted 28 Aug 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of the study was to evaluate the safety and tolerability of VX-809 in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
Detailed description
This was a Phase 2, randomized, double-blind, placebo-controlled, multiple-dose study of orally-administered VX-809 in participants with CF who are homozygous for the specific CFTR mutation known as ∆F508 or F508del. Enrollment was planned for 90 participants at approximately 20 centers. Participants were planned to be randomized in a 4:1 ratio to receive 1 of 4 doses of VX-809 or placebo once a day for 28 days in a parallel design. Participants were outpatients during the study, except for overnight stays on Day 1 and 28.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboPlacebo matched to VX-809 capsule orally once daily for 28 days. |
| VX-809, 25 mg | Experimental | Drug: VX-809VX-809, 25 milligram (mg) capsule orally once daily for 28 days. |
| VX-809, 50 mg | Experimental | Drug: VX-809VX-809, 50 mg capsule orally once daily for 28 days. |
| VX-809, 100 mg | Experimental | Drug: VX-809VX-809, 100 mg capsule orally once daily for 28 days. |
| VX-809, 200 mg | Experimental | Drug: VX-809VX-809, 200 mg capsule orally once daily for 28 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Confirmed diagnosis of CF with ∆F508-CFTR mutation in both alleles
- Forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent (%) of predicted normal for age, gender, and height
- Weight >=40 kilograms (kg) and body mass index greater than or equal to 18.5 kilogram per square meter (kg/m^2)
- Screening laboratory values, tests, and physical examination within acceptable ranges
- Negative pregnancy test (for women of child-bearing potential)
Key exclusion criteria (4 listed)
- History of any illness, or any ongoing acute illness, that could impact the safety of the study participant or may confound results of study
- Pulmonary exacerbation or changes in therapy for pulmonary disease within 14 days before receiving the first dose of study drug
- Impaired hepatic or renal function
- History of organ or hematological transplant
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 |
|---|---|---|
| VX-809Capsules | Drug | VX-809, 100 mg, VX-809, 200 mg, VX-809, 25 mg, VX-809, 50 mg |
| PlaceboPlacebo matched to VX-809 capsules. | Drug | Placebo |
Primary outcome
Safety and Tolerability Based on Adverse Events (AEs)
Time frame: Up to 14 days after last dose (last dose = Day 28)
AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. Serious adverse event (SAE) (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Number of participants with AEs and SAEs are reported. An AE that started at or after initial dosing of study drug, or increased in severity after initial dosing of study drug visit is considered treatment-emergent.
9 secondary outcomes
Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Day 28
Time frame: Baseline, Day 28
Change From Baseline in Percent Predicted FEV1 at Day 28
Time frame: Baseline, Day 28
Change From Baseline in Forced Vital Capacity (FVC) at Day 28
Time frame: Baseline, Day 28
Change From Baseline in Forced Expiratory Flow Over the Middle Half of the FVC (FEF25-75) at Day 28
Time frame: Baseline, Day 28
Change From Baseline in Sweat Chloride at Day 28
Time frame: Baseline, Day 28
Change From Baseline in Nasal Potential Difference (NPD) of Zero Chloride Plus Isoproterenol Response at Day 28
Time frame: Baseline, Day 28
Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Domain Scores at Day 28
Time frame: Baseline, Day 28
Maximum Plasma Concentration (Cmax) of VX-809
Time frame: Day 1 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post-dose), Day 28 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, 24, and 30-60 hours post dose)
Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24) of VX-809
Time frame: Day 1 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post-dose), Day 28 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post dose)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Aug 2015.
- 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.
- Derived from this study: PubMed 21825083Clancy JP, Rowe SM, Accurso FJ, Aitken ML, Amin RS, Ashlock MA, Ballmann M, Boyle MP, Bronsveld I, Campbell PW, De Boeck K, Donaldson SH, Dorkin HL, Dunitz JM, Durie PR, Jain M, Leonard A, McCoy KS, Moss RB, Pilewski JM, Rosenbluth DB, Rubenstein RC, Schechter MS, Botfield M, Ordonez CL, Spencer-Green GT, Vernillet L, Wisseh S, Yen K, Konstan MW. Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation. Thorax. 2012 Jan;67(1):12-8. doi: 10.1136/thoraxjnl-2011-200393. Epub 2011 Aug 8.
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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.
Medical Monitor study director
Vertex Pharmaceuticals Incorporated
25 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VX-809 in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 25 sites in 5 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 7 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 28 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States, Belgium, Germany, Netherlands, Canada. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 28 Aug 2015). 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?
Safety and Tolerability Based on Adverse Events (AEs) (time frame: Up to 14 days after last dose (last dose = Day 28)). 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 NCT00865904, which also lists 3 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 28 Aug 2015.