Cystic fibrosis subjects Homozygous for the F508del-CFTR Mutation MedDRA version: 19.1 Level: SOC Classification code 10010331 Term: Congenital, familial and genetic disorders System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Orkambi
Product Name: Orkambi
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Lumacaftor
Current Sponsor code: VX-809
VRT-826809
VRT-0826809
Other descriptive name: LUMACAFTOR
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 200 -
INN or Proposed INN: IVACAFTOR
CAS Number: 873054-44-5
Current
Sponsors
Vertex Pharmaceuticals Incoporated
Eligibility
Sex/Gender
All
Inclusion criteria
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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the impact of smart adherence technology for monitoring LUM/IVA adherence rates among subjects 16 years of age and older with CF who are homozygous for the F508del-CFTR mutation.;Secondary Objective: To collect subject and physician feedback on the use of smart adherence technology to monitor LUM/IVA adherence;Primary end point(s): Mean percentage adherence to LUM/IVA treatment over 48 weeks;Timepoint(s) of evaluation of this end point: 48 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Mean percentage adherence to LUM/IVA treatment over 24 weeks 2. Mean percentage adherence to LUM/IVA treatment from Week 25 through Week 48 3. Proportion of subjects with =80% LUM/IVA adherence over 24 weeks 4. Proportion of subjects with =80% LUM/IVA adherence over 48 weeks 5. Proportion of subjects with =80% LUM/IVA adherence from Week 25 through Week 48 6. Proportion of subjects with a non-physician-directed LUM/IVA interruption =72 hours over 48 weeks 7. Number of non-physician-directed LUM/IVA interruptions =72 hours over 48 weeks 8. Time to the first non-physician-directed LUM/IVA interruption =72 hours over 48 weeks;Timepoint(s) of evaluation of this end point: 1. Over 24 weeks 2. From Week 25 through Week 48 3. Over 24 weeks 4. Over 48 weeks 5. From Week 25 through Week 48 6. Over 48 weeks 7. Over 48 weeks 8. Over 48 weeks | — |
Countries
Australia, Canada, Spain, United Kingdom, United States
Contacts
Public ContactClinical Trials and Medical Info
Vertex Pharmaceuticals Incorporated
Outcome results
None listed