Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation Clinical Trial
Official title:
A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
The purpose of this study is to evaluate the efficacy and safety of long-term treatment with lumacaftor in combination with ivacaftor in people 12 years and older with Cystic Fibrosis.
| Status | Completed |
| Enrollment | 1165 |
| Est. completion date | April 2016 |
| Est. primary completion date | April 2016 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | 12 Years and older |
| Eligibility |
Inclusion Criteria: - Signed informed consent form (ICF), and where appropriate, signed assent form. - Subjects entering the Part A Treatment Cohort: Completed 24 weeks of study drug treatment in Study 103 or Study 104 and elect to enroll in Part A treatment cohort. - Subjects entering the Part B Treatment Cohort: Completed 56 days of study drug treatment in Cohort 4 of Study 102 and elect to enroll in Part B treatment cohort. - Subjects entering the Part A Observational Cohort: Completed 24 weeks of study drug treatment in Study 103 or Study 104, but do not elect to enroll in the Part A Treatment Cohort or do not qualify to enroll in Part A treatment cohort. - Willing to remain on a stable CF medication regimen through the end of study (Part A and Part B Treatment Cohorts only). Exclusion Criteria: - Any comorbidity or laboratory abnormality that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject (e.g., cirrhosis with portal hypertension). - Pregnant and nursing females. Females of childbearing potential must have a negative pregnancy test at the Day 1 Visit. - History of drug intolerance in the prior study that would pose an additional risk to the subject in the opinion of investigator or Vertex. - History of poor compliance with study drug and/or procedures in the previous study as deemed by the investigator. - Participation in an investigational drug trial (including studies investigating lumacaftor and/or ivacaftor, or studies requiring blood collections with or without administration of study drug) |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| n/a | |||
| Lead Sponsor | Collaborator |
|---|---|
| Vertex Pharmaceuticals Incorporated |
United States, Australia, Austria, Belgium, Canada, Czech Republic, Denmark, France, Germany, Ireland, Italy, Netherlands, Spain, Sweden, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Treatment cohorts: safety of long term treatment based on adverse events (AEs), clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), vital signs, and pulse oximetry | through 100 weeks | Yes | |
| Secondary | Absolute change from baseline in percent predicted FEV1 | baseline through 100 weeks | No | |
| Secondary | Relative change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) | baseline through 100 weeks | No | |
| Secondary | Absolute change from baseline in Cystic Fibrosis Questionnaire - Revised (CFQ R) respiratory domain score | baseline through 100 weeks | No | |
| Secondary | Absolute change from baseline in body mass index (BMI) | baseline through 100 weeks | No | |
| Secondary | Number of pulmonary exacerbations starting from the previous study (Part A only) | previous study through 100 weeks | No | |
| Secondary | Absolute change in BMI z score (Part A only) | baseline through 100 weeks | No | |
| Secondary | Absolute change from baseline in body weight | baseline through 100 weeks | No | |
| Secondary | Time to first pulmonary exacerbation, including pulmonary exacerbations in the previous study (Part A only) | baseline through 100 weeks | No | |
| Secondary | Event of having at least 1 pulmonary exacerbation, including pulmonary exacerbations in the previous study (Part A only) | baseline through 100 weeks | No | |
| Secondary | Rate of change in percent predicted FEV1 | baseline through 100 weeks | No | |
| Secondary | Number of pulmonary exacerbations starting from the current study (Part A) | baseline through 100 weeks | No | |
| Secondary | Time-to-first pulmonary exacerbation in the current study (Part A only) | baseline through 100 weeks | No | |
| Secondary | Event of having at least 1 pulmonary exacerbation in the current study (Part A only) | baseline through 100 weeks | No | |
| Secondary | Part A Observational Cohort: Safety, as determined by Serious Adverse Events | up to 2 years | No |