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 Mutatio
- Conditions
- Cystic Fibrosis10083624
- Registration Number
- NL-OMON41659
- Lead Sponsor
- Vertex Pharmaceuticals
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 28
Subjects who meet all of the following inclusion criteria will be eligible for this study.
1. Signed informed consent form (ICF), and where appropriate, signed assent form.
2. Subjects entering the Part A Treatment Cohort must meet both of the following
criteria:
* Completed 24 weeks of study drug treatment in Study 103 or Study 104
* Subjects who had study drug interruptions, but completed study visits up to
Week 24 of Study 103 or 104 are eligible. Subjects who are not taking study drug
at the Week 24 Visit, including subjects that require study drug interruption to be either continued or initiated at Day 1 in Study 105, must have received Vertex approval for enrollment/randomization in the Part A Treatment Cohort.
* Elect to enroll in the Part A Treatment Cohort
Subjects entering the Part A Observational Cohort must meet the following criteria:
* 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
* Subjects who received at least 4 weeks of study drug and completed visits up to
Week 24 Visit of Study 103 or 104 but are not taking study drug at the Week 24
Visit because of a drug interruption and did not receive Vertex approval for
enrollment into the Part A Treatment Cohort (or elect not to enroll in the Part A
Treatment Cohort).
* Subjects who permanently discontinued study drug after receiving at least
4 weeks of study drug and remained in the study from the time of discontinuation
of study drug treatment through the Week 24 Visit in Study 103 or Study 104
Subjects entering the Part B Treatment Cohort must meet both of the following
criteria:
* Completed 56 days of study drug treatment in Cohort 4 of Study 102
* Subjects who had study drug interruptions but completed study visits up to
Day 56 are eligible. Subjects who are not taking study drug at the Day 56 Visit, including subjects that require study drug interruption to be either continued or initiated at Day 1 in Study 105,
must have received Vertex approval for enrollment/randomization in the Part B Treatment
Cohort.
* Elect to enroll in the Part B Treatment Cohort
3. Willing to remain on a stable CF medication regimen through the end of study (Part A
and Part B Treatment Cohorts only).
4. Able to understand and comply with protocol requirements, restrictions, and instructions,
and likely to complete the study as planned, as judged by the investigator and Vertex,
based in part on study compliance in Study 103, Study 104, and Cohort 4 of Study 102.
Subjects who meet any of the following exclusion criteria will NOT be eligible for this study.
1. 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).
2. Pregnant and nursing females. Females of childbearing potential must have a negative
urine pregnancy test at the Day 1 Visit (enrollment/randomization) and before recieving the first dose of study drug (Section 12.7.2).
3. Sexually active subjects of reproductive potential who are not willing to follow the
contraception requirements outlined in Section 12.7.7. of the protocol
4. History of drug intolerance in the prior study that would pose an additional risk to the
subject in the opinion of investigator or Vertex. Examples of subjects who may not be
eligible for any of the treatment arms include the following:
* Subjects with a history of allergy or hypersensitivity to the study drug
* Liver function test (LFT) abnormality during study drug treatment in the previous
study (Study 103, Study 104, or Cohort 4 of Study 102) for which a clear cause was not identified.
* Other severe or life-threatening reactions to the study drug in the previous study
5. History of poor compliance with study drug and/or procedures in the previous study as
deemed by the investigator.
6. Participation in an investigational drug trial (including studies investigating lumacaftor
and/or ivacaftor. NOTE: participation in a noninterventional study (including observational studies and studies requiring blood collections without administration of study drug is permitted.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Part A and Part B Treatment Cohorts:<br /><br>Safety of long-term treatment of lumacaftor in combination with ivacaftor based<br /><br>on adverse events (AEs), clinical laboratory values (serum chemistry,<br /><br>hematology, coagulation studies, and urinalysis), standard digital<br /><br>electrocardiograms (ECGs), vital signs, and pulse oximetry<br /><br><br /><br>Part A Observational Cohort:<br /><br>Not applicable.</p><br>
- Secondary Outcome Measures
Name Time Method