A Pilot Study to Evaluate the Use of Smart Adherence Technology to Measure Lumacaftor/Ivacaftor Adherence in CF Subjects Homozygous for the F508del-CFTR Mutatio

Phase 1

• Conditions: Cystic fibrosis subjects Homozygous for the F508del-CFTR Mutation; MedDRA version: 19.1Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2016-004996-33-ES
• Lead Sponsor: Vertex Pharmaceuticals Incoporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-03-10
• Last Update Posted: 3 July 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 75

Inclusion Criteria

1. Subject and/or legally appointed and authorized representative (e.g., parents or legal guardian) will sign and date an informed consent form (ICF) and where appropriate, an assent form.
2. Subject and/or legally appointed and authorized representative (e.g., parent or legal guardian) is willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
3. Confirmed diagnosis of CF and homozygous for the F508del-CFTR mutation at Screening.
4. Subjects (male and female) will be aged 16 years and older on the date of informed consent or, where appropriate, assent.
5. FEV1 =40% of predicted normal for age, sex, and height at Screening.
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 60
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering LUM/IVA to the subject, e.g., history of advanced liver disease.
2. Presence of moderate or severe hepatic impairment (Child-Pugh Class B or C).
3. Subjects currently receiving invasive mechanical ventilation.
4. Known history of alcohol or drug abuse in the past year, including but not limited to cannabis, cocaine, and opiates, as deemed by the investigator.
5. Any of the following abnormal laboratory values during screening:
o Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >5 × ULN o ALT or AST >3 × ULN with total bilirubin >2 × ULN o Glomerular filtration rate =30 mL/min/1.73 m2. This will be calculated by the Modification of Diet in Renal Disease study equation for subjects =18 years of age and calculated by the Counahan-Barratt equation for subjects aged 12 to 17 years.
6. Pregnant or nursing females (females of childbearing potential must have a negative pregnancy test at screening and Day 1).
7. 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 LUM/IVA.
8. Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements outlined in Section 11.5.7.1.
9. History of solid organ or hematological transplantation.
10. Ongoing or prior participation in an investigational drug study (including studies
investigating LUM and/or IVA) within 30 days of screening
- A washout period of 5 terminal half-lives of the previous investigational LUM/IVA or 30 days, whichever is longer
- The duration of the elapsed time may be longer if required by local regulations.
11. Current use of commercial LUM/IVA combination therapy.
12. Subject, or close relative of the subject, is the investigator or a subinvestigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified