A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
- Conditions
- Cystic fibrosisMedDRA version: 20.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
- Registration Number
- EUCTR2019-000833-37-NL
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 250
Part A
Subjects transitioning from Study 104 must meet all of the following
criteria to be eligible for Part A:
1. Subject (or his or her legally appointed and authorized representative) will sign and date an ICF (informed consent form), and, when appropriate, an assent form.
2. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
3. Did not withdraw consent from the parent study.
4. Meets at least 1 of the following criteria:
- Completed study drug treatment in the parent study.
- Had study drug interruption(s) in the parent study, but without prematurely discontinuing study drug in the parent study and completed study visits up to the last scheduled visit of the Treatment Period of the parent study.
5. Willing to remain on a stable CF treatment regimen through completion of study participation.
Inclusion criteria for subjects resuming participation in Part A of this
study after enrolling in another qualifying Vertex study include all of the
criteria above AND the following criterion:
6. Completed the ETT visit in another qualified Vertex study before or on
same day as the Returning Visit in this study. If more than 30 days have
elapsed since the ETT visit in the other qualified Vertex study, approval
of the medical monitor is required.
Part B
Subjects who meet all of the following criteria will be eligible for Part B:
1. Subject (or his or her legally appointed and authorized
representative) will sign and date an ICF, and, when appropriate, an
assent form.
2. Willing and able to comply with scheduled visits, treatment plan,
study restrictions, laboratory tests, contraceptive guidelines, and other
study procedures.
3. Did not withdraw consent from the parent study or Part A of this
study.
4. Meets at least 1 of the following criteria:
• Completed study drug treatment in Part A, or
• Had study drug interruption(s) in Part A, but without prematurely
discontinuing study drug and completed study visits up to the last
scheduled visit of the Treatment Period of Part A.
5. Willing to remain on a stable CF treatment regimen (as defined in
Protocol Section 9.5) through completion of study participation.
Inclusion criteria for subjects resuming participation in Part B of this
study after enrolling in another qualifying Vertex study include all of the
criteria above AND the following criterion:
6. Completed the ETT visit in another qualified Vertex study before or on
same day as the Returning Visit in this study. If more than 30 days have
elapsed since the ETT visit in the other qualified Vertex study, approval
of the medical monitor is required.
Are the trial subjects under 18? yes
Number of subjects for this age range: 75
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 175
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Part A
Subjects who meet any of the following criteria will NOT be eligible for
Part A:
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 study drug to the subject.
2. Pregnant and breast-feeding females. All female subjects, regardless of childbearing potential status, must have a negative pregnancy test at the Part A Day 1 Visit before receiving the first dose of study drug.
3. History of intolerance to study drug in the parent study that would pose an additional risk to the subject in the opinion of the investigator. (e.g., subjects with a history of allergy or hypersensitivity to the study drug).
4. Current participation in an investigational drug study (other than the parent study). Participation in a non-interventional study (including observational studies, registry studies, and studies requiring blood collections without administration of study drug) and screening for another Vertex study is permitted.
Subjects resuming participation in Part A of this study after enrolling in
another qualifying Vertex study must not meet Exclusion Criteria 1
through 3, and also must not meet any of the following criteria:
5. Pregnant and nursing females. All female subjects must have a
negative pregnancy test at the Returning Visit before receiving the first
dose of study drug.
6. History of intolerance to study drug that would pose an additional risk
to the subject in the opinion of the investigator. If protocol-defined
interruption criteria is met in another qualified
Vertex study, then the subject must remain on study drug interruption
until the subject meets criteria in Protocol Section 9.8 and receives
approval by the medical monitor to resume study drug dosing in this
study.
7. Has received the first dose of study drug in the Treatment Period of
another qualified Vertex study.
8. Has access to commercially available VX-445/TEZ/IVA or is receiving
managed-access-program-supplied VX-445/TEZ/IVA.
9. Has departed this study more than once to participate in another
qualified Vertex study.
Part B
Subjects who meet any of the following criteria will NOT be eligible for
this study:
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 study drug to the subject.
2. Pregnant and breast-feeding females. All female subjects, regardless
of childbearing potential status, must have a negative pregnancy test at
the Part B Day 1 Visit before receiving the first dose of study drug.
3. History of intolerance to study drug that would pose an additional risk
to the subject in the opinion of the investigator (e.g., subjects with a
history of allergy or hypersensitivity to the study drug).
4. Current participation in an investigational drug study (other than the
parent study or the current study). Participation in a non-interventional
study (including observational studies, registry studies, and studies
requiring blood collections without administration of study drug) and
screening for another Vertex study is permitted.
Subjects resuming participation in Part B of this study after enrolling in
another qualifying Vertex study must not meet Exclusion Criteria 1
through 4, and also must not meet any of the following criteria:
5. Pregnant and nursing females. All female subjects must have a
negative pregnancy test at
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method