Study Stopped
Study terminated early due to slow recruitment of patients.
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, 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
1 other identifier
interventional
24
2 countries
11
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_4
Started Jun 2016
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 1, 2016
CompletedFirst Submitted
Initial submission to the registry
July 1, 2016
CompletedFirst Posted
Study publicly available on registry
July 6, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2017
CompletedResults Posted
Study results publicly available
September 27, 2018
CompletedSeptember 27, 2018
August 1, 2018
1.2 years
July 1, 2016
August 31, 2018
August 31, 2018
Conditions
Outcome Measures
Primary Outcomes (1)
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment
Percentage adherence was reported in terms of median and full range due to small sample size.
Up to 35 weeks
Secondary Outcomes (3)
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment Through 12 Weeks
Up to Week 12
Number of Participants With Greater Than or Equal to (>=) 80 Percent (%) Adherence
Up to Week 12
Number of Participants With Greater Than or Equal to (>=) 90 Percent (%) Adherence
Up to Week 12
Study Arms (2)
Arm A: activated smart device alerts and feedback
EXPERIMENTALLUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
Arm B: de-activated smart device alerts/feedback features
EXPERIMENTALLUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
Interventions
LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (11)
Unknown Facility
Hartford, Connecticut, United States
Unknown Facility
Washington D.C., District of Columbia, United States
Unknown Facility
Orlando, Florida, United States
Unknown Facility
Atlanta, Georgia, United States
Unknown Facility
Jackson, Mississippi, United States
Unknown Facility
Albuquerque, New Mexico, United States
Unknown Facility
Columbia, South Carolina, United States
Unknown Facility
Edmonton, Alberta, Canada
Unknown Facility
Victoria, British Columbia, Canada
Unknown Facility
Saint John, New Brunswick, Canada
Unknown Facility
Québec, Canada
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Limitations and Caveats
As a result of the early termination, definitive conclusions cannot be made given the limited sample size and incomplete data.
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 1, 2016
First Posted
July 6, 2016
Study Start
June 1, 2016
Primary Completion
August 1, 2017
Study Completion
August 1, 2017
Last Updated
September 27, 2018
Results First Posted
September 27, 2018
Record last verified: 2018-08