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

Vertex Pharmaceuticals

Status and phase: Terminated; Phase 4

Conditions: Cystic Fibrosis

Treatments: Device: de-activated smart device; Drug: LUM/IVA; Device: activated smart device

Study type: Interventional

Funder types: Industry

Identifiers

NCT02823470

VX15-809-114

Details and patient eligibility

About

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.

Enrollment: 24 patients

Sex: All

Ages: 16+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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24 participants in 2 patient groups

Arm A: activated smart device alerts and feedback

Experimental group

Description:

LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.

Treatment:

Drug: LUM/IVA

Device: activated smart device

Arm B: de-activated smart device alerts/feedback features

Experimental group

Description:

LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.

Treatment:

Drug: LUM/IVA

Device: de-activated smart device