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

August 31, 2018 updated by: Vertex Pharmaceuticals Incorporated

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

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

24

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
      • Quebec, Canada
    • Alberta
      • Edmonton, Alberta, Canada
    • British Columbia
      • Victoria, British Columbia, Canada
    • New Brunswick
      • Saint John, New Brunswick, Canada
  • United States
    • Connecticut
      • Hartford, Connecticut, United States
    • District of Columbia
      • Washington, District of Columbia, United States
    • Florida
      • Orlando, Florida, United States
    • Georgia
      • Atlanta, Georgia, United States
    • Mississippi
      • Jackson, Mississippi, United States
    • New Mexico
      • Albuquerque, New Mexico, United States
    • South Carolina
      • Columbia, South Carolina, United States

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Arm A: activated smart device alerts and feedback
LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
Drug: LUM/IVA
LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
Other Names:
  • lumacaftor/ivacaftor
Device: activated smart device
Experimental: Arm B: de-activated smart device alerts/feedback features
LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
Drug: LUM/IVA
LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
Other Names:
  • lumacaftor/ivacaftor
Device: de-activated smart device

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment
Time Frame: Up to 35 weeks
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
Time Frame: Up to Week 12
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
Time Frame: Up to Week 12
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
Time Frame: Up to Week 12
Number of participants with >=90% adherence to LUM/IVA treatment over 12 weeks were reported.
Up to Week 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2016

Primary Completion (Actual)

August 1, 2017

Study Completion (Actual)

August 1, 2017

Study Registration Dates

First Submitted

July 1, 2016

First Submitted That Met QC Criteria

July 5, 2016

First Posted (Estimate)

July 6, 2016

Study Record Updates

Last Update Posted (Actual)

September 27, 2018

Last Update Submitted That Met QC Criteria

August 31, 2018

Last Verified

August 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX15-809-114

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Cystic Fibrosis

Clinical Trials on LUM/IVA

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Ireland

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe