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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: LUM/IVA
* Device: activated smart device
* Device: de-activated smart device


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 16 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Arm A: activated smart device alerts and feedback<br>LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48. | Drug: LUM/IVA<br>* LUM 400 mg q12h/IVA 250 mg q12h through Week 48.<br>* Other names: lumacaftor/ivacaftor;Device: activated smart device<br> <br> |
| Experimental: Arm B: de-activated smart device alerts/feedback features<br>LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48. | Drug: LUM/IVA<br>* LUM 400 mg q12h/IVA 250 mg q12h through Week 48.<br>* Other names: lumacaftor/ivacaftor;Device: de-activated smart device<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | 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. | Up to Week 12 |