Clinical trial NCT02823470: 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
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2016-07-06
Enrollment: 24
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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
* 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

Primary outcomes:
measure: Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment
timeFrame: Up to 35 weeks
Secondary outcomes:
measure: Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment Through 12 Weeks
timeFrame: Up to Week 12
measure: Number of Participants With Greater Than or Equal to (>=) 80 Percent (%) Adherence
timeFrame: Up to Week 12
measure: Number of Participants With Greater Than or Equal to (>=) 90 Percent (%) Adherence
timeFrame: Up to Week 12
Other outcomes:
Locations:
city: Hartford
country: United States
state: Connecticut
city: Washington
country: United States
state: District of Columbia
city: Orlando
country: United States
state: Florida
city: Atlanta
country: United States
state: Georgia
city: Jackson
country: United States
state: Mississippi
city: Albuquerque
country: United States
state: New Mexico
city: Columbia
country: United States
state: South Carolina
city: Edmonton
country: Canada
state: Alberta
city: Victoria
country: Canada
state: British Columbia
city: Saint John
country: Canada
state: New Brunswick
city: Quebec
country: Canada
Study officials:
References: