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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

Primary Purpose

Cystic Fibrosis

Status
Terminated
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
LUM/IVA
activated smart device
de-activated smart device
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

16 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Arm A: activated smart device alerts and feedback

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

Arm Description

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

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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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.
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.
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.

Full Information

First Posted
July 1, 2016
Last Updated
August 31, 2018
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT02823470
Brief Title
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
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2018
Overall Recruitment Status
Terminated
Why Stopped
Study terminated early due to slow recruitment of patients.
Study Start Date
June 2016 (undefined)
Primary Completion Date
August 2017 (Actual)
Study Completion Date
August 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
24 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Arm A: activated smart device alerts and feedback
Arm Type
Experimental
Arm Description
LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
Arm Title
Arm B: de-activated smart device alerts/feedback features
Arm Type
Experimental
Arm Description
LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
Intervention Type
Drug
Intervention Name(s)
LUM/IVA
Other Intervention Name(s)
lumacaftor/ivacaftor
Intervention Description
LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
Intervention Type
Device
Intervention Name(s)
activated smart device
Intervention Type
Device
Intervention Name(s)
de-activated smart device
Primary Outcome Measure Information:
Title
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment
Description
Percentage adherence was reported in terms of median and full range due to small sample size.
Time Frame
Up to 35 weeks
Secondary Outcome Measure Information:
Title
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment Through 12 Weeks
Description
Percentage adherence was reported in terms of median and full range due to small sample size.
Time Frame
Up to Week 12
Title
Number of Participants With Greater Than or Equal to (>=) 80 Percent (%) Adherence
Description
Number of participants with >=80% adherence to LUM/IVA treatment over 12 weeks were reported.
Time Frame
Up to Week 12
Title
Number of Participants With Greater Than or Equal to (>=) 90 Percent (%) Adherence
Description
Number of participants with >=90% adherence to LUM/IVA treatment over 12 weeks were reported.
Time Frame
Up to Week 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
City
Hartford
State/Province
Connecticut
Country
United States
City
Washington
State/Province
District of Columbia
Country
United States
City
Orlando
State/Province
Florida
Country
United States
City
Atlanta
State/Province
Georgia
Country
United States
City
Jackson
State/Province
Mississippi
Country
United States
City
Albuquerque
State/Province
New Mexico
Country
United States
City
Columbia
State/Province
South Carolina
Country
United States
City
Edmonton
State/Province
Alberta
Country
Canada
City
Victoria
State/Province
British Columbia
Country
Canada
City
Saint John
State/Province
New Brunswick
Country
Canada
City
Quebec
Country
Canada

12. IPD Sharing Statement

Learn more about this trial

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

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