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

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02823470
• Other study ID #: VX15-809-114
• Status: Terminated
• Phase: Phase 4
• Start date: June 2016
• Est. completion date: August 2017

Study information

• Verified date: August 2018
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 24
• Est. completion date: August 2017
• Est. primary completion date: August 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis

Intervention

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

Device:
• activated smart device

• de-activated smart device

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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