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

Phase 4

Terminated

• Conditions: Cystic Fibrosis
• Interventions: Drug: LUM/IVA; Device: activated smart device; Device: de-activated smart device

• Registration Number: NCT02823470
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-06
• Study First Submitted: 2016-07-01
• Study First Submitted QC: 2016-07-05
• Study First Posted: 2016-07-06
• Primary Completion: 2017-08
• Study Completion: 2017-08
• Status Verified: 2018-08
• Results First Submitted: 2018-08-31
• Results First Submitted QC: 2018-08-31
• Last Update Submitted: 2018-08-31
• Results First Posted: 2018-09-27
• Last Update Posted: 2018-09-27

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 24

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Arm A: activated smart device alerts and feedback	activated smart device	LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
Arm A: activated smart device alerts and feedback	LUM/IVA	LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
Arm B: de-activated smart device alerts/feedback features	LUM/IVA	LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
Arm B: de-activated smart device alerts/feedback features	de-activated smart device	LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.

Primary Outcome Measures

Name	Time	Method
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment	Up to 35 weeks	Percentage adherence was reported in terms of median and full range due to small sample size.

Secondary Outcome Measures

Name	Time	Method
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment Through 12 Weeks	Up to Week 12	Percentage adherence was reported in terms of median and full range due to small sample size.
Number of Participants With Greater Than or Equal to (>=) 80 Percent (%) Adherence	Up to Week 12	Number of participants with \>=80% adherence to LUM/IVA treatment over 12 weeks were reported.
Number of Participants With Greater Than or Equal to (>=) 90 Percent (%) Adherence	Up to Week 12	Number of participants with \>=90% adherence to LUM/IVA treatment over 12 weeks were reported.