Cystic FibrosisPhase 4NCT02823470
A Pilot Study to Evaluate the Use of Smart Adherence Technology to Measure Lumacaftor/Ivacaftor Adherence in CF Subjects Homozygous for the F508del CFTR Mutation
A Phase 4 study testing LUM/IVA, activated smart device and de-activated smart device in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 11 sites in 2 countries.
Trial summary
The study will evaluate the impact of smart adherence technology for monitoring on lumacaftor/ivacaftor (LUM/IVA) adherence rates among subjects 16 years of age and older with Cystic Fibrosis (CF) who are homozygous for the F508del Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) mutation. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- LUM/IVA, activated smart device and de-activated smart device
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 11 sitesin 2 countries
- Study dates
- Jun 2016to Aug 2017
- Phase
- Phase 4
- 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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject (or his or her legally appointed and authorized representative) will sign and date an informed consent form (ICF).
- Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
- Confirmed diagnosis of CF and homozygous for the F508del-CFTR mutation
- Forced Expiratory Volume in one second/forced vital capacity (FEV1) ≥40% of predicted normal for age, sex, and height
Who cannot join
- Presence of moderate or severe hepatic impairment (Child-Pugh Class B or C)
- Subjects currently receiving invasive mechanical ventilation
- Known history of alcohol or drug abuse in the past year
- Clinically significant abnormal laboratory values during screening
- Pregnant or nursing females
- Female subjects and female partners of male subjects who plan to become pregnant during Treatment Period or within 90 days following the last dose of study drug
Show all 9Show fewer
- History of solid organ or hematological transplantation
- Ongoing or prior participation in an investigational drug study within 30 days of screening
- Current use of commercial LUM/IVA combination therapy
Trial locations and contacts
11 sites in 2 countries.
11 sites in 2 countries
United States7 sites
Study site
Albuquerque, New Mexico
Study site
Atlanta, Georgia
Study site
Columbia, South Carolina
Study site
Hartford, Connecticut
Study site
Jackson, Mississippi
Study site
Orlando, Florida
Study site
Washington D.C., District of Columbia
Canada4 sites
Study site
Edmonton, Alberta
Study site
Québec
Study site
Saint John, New Brunswick
Study site
Victoria, British Columbia
About this study
Official title: A Phase 4, Open-label Treatment, Randomized, Multicenter, 2-arm, Parallel-group, Pilot Study of Adherence to Lumacaftor/Ivacaftor in CF Subjects Homozygous for the F508del CFTR Mutation
The study will evaluate the impact of smart adherence technology for monitoring on lumacaftor/ivacaftor (LUM/IVA) adherence rates among subjects 16 years of age and older with Cystic Fibrosis (CF) who are homozygous for the F508del Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) mutation.
- Conditions
- Cystic Fibrosis
- Being tested
- LUM/IVA, activated smart device, de-activated smart device
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LUM/IVADrug
Also called lumacaftor/ivacaftor
LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
activated smart deviceDevice
de-activated smart deviceDevice
| Study group | Role | What they receive |
|---|---|---|
| Arm A: activated smart device alerts and feedback | Experimental | LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48. Receives: Drug: LUM/IVA; Device: activated smart device |
| Arm B: de-activated smart device alerts/feedback features | Experimental | LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48. Receives: Drug: LUM/IVA; Device: de-activated smart device |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment
Percentage adherence was reported in terms of median and full range due to small sample size.
Measured: Up to 35 weeks
Secondary outcomes (3)
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment Through 12 Weeks
Percentage adherence was reported in terms of median and full range due to small sample size.
Measured: Up to Week 12
Number of Participants With Greater Than or Equal to (>=) 80 Percent (%) Adherence
Number of participants with >=80% adherence to LUM/IVA treatment over 12 weeks were reported.
Measured: Up to Week 12
Number of Participants With Greater Than or Equal to (>=) 90 Percent (%) Adherence
Number of participants with >=90% adherence to LUM/IVA treatment over 12 weeks were reported.
Measured: 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 posted6 Jul 2016 (estimated)
- Study startJun 2016
- Primary completionAug 2017
- Study completionAug 2017
Why it stopped: Study terminated early due to slow recruitment of patients.
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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 24 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
- NCT02823470
- Sponsor's protocol ID
- VX15-809-114
- Status last verified
- Aug 2018
- First submitted
- 1 Jul 2016
- Last updated
- 27 Sep 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 4, Open-label Treatment, Randomized, Multicenter, 2-arm, Parallel-group, Pilot Study of Adherence to Lumacaftor/Ivacaftor in CF Subjects Homozygous for the F508del CFTR Mutation
- ClinicalTrials.gov ID
- NCT02823470
- Acronym / sponsor ID
- VX15-809-114
- Conditions
- Cystic Fibrosis
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study terminated early due to slow recruitment of patients.
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- Jun 2016
- Primary completion
- Aug 2017 (actual)
- Study completion
- Aug 2017 (actual)
- First posted
- 6 Jul 2016 (estimated)
- Results
- Posted 27 Sep 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study will evaluate the impact of smart adherence technology for monitoring on lumacaftor/ivacaftor (LUM/IVA) adherence rates among subjects 16 years of age and older with Cystic Fibrosis (CF) who are homozygous for the F508del Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) mutation.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm A: activated smart device alerts and feedback | Experimental | Drug: LUM/IVA, Device: activated smart deviceLUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48. |
| Arm B: de-activated smart device alerts/feedback features | Experimental | Drug: LUM/IVA, Device: de-activated smart deviceLUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Subject (or his or her legally appointed and authorized representative) will sign and date an informed consent form (ICF).
- Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
- Confirmed diagnosis of CF and homozygous for the F508del-CFTR mutation
- Forced Expiratory Volume in one second/forced vital capacity (FEV1) ≥40% of predicted normal for age, sex, and height
Key exclusion criteria (9 listed)
- Presence of moderate or severe hepatic impairment (Child-Pugh Class B or C)
- Subjects currently receiving invasive mechanical ventilation
- Known history of alcohol or drug abuse in the past year
- Clinically significant abnormal laboratory values during screening
- Pregnant or nursing females
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 |
|---|---|---|
| LUM/IVAAlso: lumacaftor/ivacaftorLUM 400 mg q12h/IVA 250 mg q12h through Week 48. | Drug | Arm A: activated smart device alerts and feedback, Arm B: de-activated smart device alerts/feedback features |
| activated smart device | Device | Arm A: activated smart device alerts and feedback |
| de-activated smart device | Device | Arm B: de-activated smart device alerts/feedback features |
Primary outcome
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment
Time frame: Up to 35 weeks
Percentage adherence was reported in terms of median and full range due to small sample size.
3 secondary outcomes
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment Through 12 Weeks
Time frame: Up to Week 12
Number of Participants With Greater Than or Equal to (>=) 80 Percent (%) Adherence
Time frame: Up to Week 12
Number of Participants With Greater Than or Equal to (>=) 90 Percent (%) Adherence
Time frame: 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 27 Sep 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.
11 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing LUM/IVA, activated smart device and de-activated smart device in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 11 sites in 2 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 9 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 27 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, 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. 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?
Yes. Results are posted in the registry (first posted 27 Sep 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?
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment (time frame: Up to 35 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 27 Sep 2018.