Cystic FibrosisPhase 1NCT03486236
A Study Evaluating Safety and Pharmacokinetics of VX-440 in Combination With Tezacaftor/Ivacaftor in Healthy Adult Subjects
A Phase 1 study testing VX-440, TEZ and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 1 site in United Kingdom.
Trial summary
This study evaluated the safety and pharmacokinetics of multiple ascending doses of VX-440 in combination with tezacaftor/ivacaftor (TEZ/IVA) (triple combination [TC]) administered for 13 days to healthy male and female subjects Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- VX-440, TEZ and IVA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 16 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 20 Jul 2016to 14 Sep 2016
- Phase
- Phase 1
- 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 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Female subjects of non-childbearing potential only.
- Body mass index (BMI) of 18.0 to 31.0 kg/m2, inclusive, and a total body weight >50 kg.
- Normal pulmonary function measurements, defined as forced expiratory volume in 1 second (FEV1) and forced vital capacity (FVC) both ≥80% of their predicted value at screening.
Who cannot join
- For female subjects: Pregnant or nursing subjects.
- Glucose-6-phosphate dehydrogenase (G6PD) deficiency.
- History of hemolysis.
- Total bilirubin level >2 × ULN at Screening.
- Other protocol defined Inclusion/Exclusion criteria applied.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1, Randomized, Double Blind, Placebo Controlled, Multiple Dose Escalation Study Evaluating Safety and Pharmacokinetics of VX-440 in Combination With VX-661/Ivacaftor in Healthy Adult Subjects
This study evaluated the safety and pharmacokinetics of multiple ascending doses of VX-440 in combination with tezacaftor/ivacaftor (TEZ/IVA) (triple combination [TC]) administered for 13 days to healthy male and female subjects
- Conditions
- Cystic Fibrosis
- Being tested
- VX-440, TEZ, IVA, Matched Placebos
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-440Drug
VX-440 was administered in TC with TEZ and IVA.
TEZDrug
Also called Tezacaftor, VX-661
TEZ was administered as part of a fixed-dose combination (FDC) tablet (TEZ/IVA)
IVADrug
Also called Ivacaftor, VX-770
IVA was administered as part of a FDC tablet (TEZ/IVA) and as a mono tablet
Matched PlacebosDrug
Placebos matched to VX-440, TEZ, and IVA.
| Study group | Role | What they receive |
|---|---|---|
| Cohort C1 | Experimental | Not described Receives: Drug: VX-440; Drug: TEZ; Drug: IVA |
| Cohort C1: Triple Placebo | Placebo comparator | Not described Receives: Drug: Matched Placebos |
| Cohort C2 | Experimental | Not described Receives: Drug: VX-440; Drug: TEZ; Drug: IVA |
| Cohort C2: Triple Placebo | Placebo comparator | Not described Receives: Drug: Matched Placebos |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability were based on the number and assessment of adverse events (AEs) and serious adverse events (SAEs)
Measured: from baseline through safety follow-up visit (up to 29 days)
Secondary outcomes (3)
Maximum observed concentration (Cmax) of VX-440, TEZ and its metabolites (M1-TEZ and M2-TEZ), and IVA and its metabolites (M1-IVA and M6-IVA)
Measured: from Day 1 through Day 18
Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-440, TEZ and its metabolites (M1-TEZ and M2-TEZ), and IVA and its metabolites (M1-IVA and M6-IVA)
Measured: from Day 1 through Day 18
Observed pre-dose concentration (Ctrough) of VX-440, TEZ and its metabolites (M1-TEZ and M2-TEZ), and IVA and its metabolites (M1-IVA and M6-IVA)
Measured: from Day 1 through Day 18
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2018
- Study start20 Jul 2016
- Primary completion14 Sep 2016
- Study completion14 Sep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 16 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
- NCT03486236
- Sponsor's protocol ID
- VX16-440-002
- Other IDs
- 2016-000762-38, 2016
- Status last verified
- Mar 2018
- First submitted
- 27 Mar 2018
- Last updated
- 3 Apr 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Double Blind, Placebo Controlled, Multiple Dose Escalation Study Evaluating Safety and Pharmacokinetics of VX-440 in Combination With VX-661/Ivacaftor in Healthy Adult Subjects
- ClinicalTrials.gov ID
- NCT03486236
- Acronym / sponsor ID
- VX16-440-002
- Conditions
- Cystic Fibrosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 20 Jul 2016 (actual)
- Primary completion
- 14 Sep 2016 (actual)
- Study completion
- 14 Sep 2016 (actual)
- First posted
- 3 Apr 2018 (actual)
- 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.
This study evaluated the safety and pharmacokinetics of multiple ascending doses of VX-440 in combination with tezacaftor/ivacaftor (TEZ/IVA) (triple combination [TC]) administered for 13 days to healthy male and female subjects
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort C1 | Experimental | Drug: VX-440, Drug: TEZ, Drug: IVA |
| Cohort C1: Triple Placebo | Placebo comparator | Drug: Matched Placebos |
| Cohort C2 | Experimental | Drug: VX-440, Drug: TEZ, Drug: IVA |
| Cohort C2: Triple Placebo | Placebo comparator | Drug: Matched Placebos |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Female subjects of non-childbearing potential only.
- Body mass index (BMI) of 18.0 to 31.0 kg/m2, inclusive, and a total body weight >50 kg.
- Normal pulmonary function measurements, defined as forced expiratory volume in 1 second (FEV1) and forced vital capacity (FVC) both ≥80% of their predicted value at screening.
Key exclusion criteria (5 listed)
- For female subjects: Pregnant or nursing subjects.
- Glucose-6-phosphate dehydrogenase (G6PD) deficiency.
- History of hemolysis.
- Total bilirubin level >2 × ULN at Screening.
- Other protocol defined Inclusion/Exclusion criteria applied.
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-440VX-440 was administered in TC with TEZ and IVA. | Drug | Cohort C1, Cohort C2 |
| TEZAlso: Tezacaftor, VX-661TEZ was administered as part of a fixed-dose combination (FDC) tablet (TEZ/IVA) | Drug | Cohort C1, Cohort C2 |
| IVAAlso: Ivacaftor, VX-770IVA was administered as part of a FDC tablet (TEZ/IVA) and as a mono tablet | Drug | Cohort C1, Cohort C2 |
| Matched PlacebosPlacebos matched to VX-440, TEZ, and IVA. | Drug | Cohort C1: Triple Placebo, Cohort C2: Triple Placebo |
Primary outcome
Safety and tolerability were based on the number and assessment of adverse events (AEs) and serious adverse events (SAEs)
Time frame: from baseline through safety follow-up visit (up to 29 days)
3 secondary outcomes
Maximum observed concentration (Cmax) of VX-440, TEZ and its metabolites (M1-TEZ and M2-TEZ), and IVA and its metabolites (M1-IVA and M6-IVA)
Time frame: from Day 1 through Day 18
Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-440, TEZ and its metabolites (M1-TEZ and M2-TEZ), and IVA and its metabolites (M1-IVA and M6-IVA)
Time frame: from Day 1 through Day 18
Observed pre-dose concentration (Ctrough) of VX-440, TEZ and its metabolites (M1-TEZ and M2-TEZ), and IVA and its metabolites (M1-IVA and M6-IVA)
Time frame: from Day 1 through Day 18
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2018.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing VX-440, TEZ and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 1 site in United Kingdom.
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 5 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 3 Apr 2018. 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 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 were based on the number and assessment of adverse events (AEs) and serious adverse events (SAEs) (time frame: from baseline through safety follow-up visit (up to 29 days)). 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 3 Apr 2018.