Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Subjects 1 Year of Age and Older
EU CTIS ID: 2022-503081-74-00
What this study is testing
To evaluate the long-term safety and tolerability of VNZ/TEZ/D-IVA
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Subject, or subject’s legally appointed and authorized representative, will sign and date an ICF. Subject will sign an assent form.
- As judged by the investigator, the legal representative or guardian must be able to understand protocol requirements, restrictions, and instructions and the legal representative or guardian should be able to ensure that the subject will comply with and is likely to complete the study as planned.
- Part A: Did not withdraw consent from the parent study.
- Part A: 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 did not prematurely discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study.
- Willing to remain on a stable CF treatment regimen through completion of study participation.
- Part B (Cohort 1): Did not withdraw consent from Part A.
You likely can't join if
- 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(s) to the subject.
- New development of a comorbidity during the parent study or Part A that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject. This includes, but is not limited to, the following: • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject. •Solid organ or hematological transplantation.
- History of drug intolerance in the parent study that would pose an additional risk to the subject in the opinion of the investigator.
- Current participation in an investigational drug study other than the parent study. Participation in a noninterventional study, and/or screening for another Vertex study is permitted.
- History of poor compliance with study drug and/or procedures in the parent study as deemed by the investigator.
- The subject or a 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.
See the full eligibility criteria
Who can join
- Subject, or subject’s legally appointed and authorized representative, will sign and date an ICF. Subject will sign an assent form.
- As judged by the investigator, the legal representative or guardian must be able to understand protocol requirements, restrictions, and instructions and the legal representative or guardian should be able to ensure that the subject will comply with and is likely to complete the study as planned.
- Part A: Did not withdraw consent from the parent study.
- Part A: 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 did not prematurely discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study.
- Willing to remain on a stable CF treatment regimen through completion of study participation.
- Part B (Cohort 1): Did not withdraw consent from Part A.
- Part B (Cohort 1): Meets at least 1 of the following criteria: • Completed study drug treatment in Part A. • Had study drug interruption(s) in Part A, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of Part A.
- Part A (Cohort 2, Pancreatic Enzyme Replacement Therapy [PERT] sub study only): Meets all of the inclusion criteria for Part A, Did not withdraw consent from Part A, Currently receiving PERT
What rules you out
- 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(s) to the subject.
- New development of a comorbidity during the parent study or Part A that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject. This includes, but is not limited to, the following: • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject. •Solid organ or hematological transplantation.
- History of drug intolerance in the parent study that would pose an additional risk to the subject in the opinion of the investigator.
- Current participation in an investigational drug study other than the parent study. Participation in a noninterventional study, and/or screening for another Vertex study is permitted.
- History of poor compliance with study drug and/or procedures in the parent study as deemed by the investigator.
- The subject or a 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.
- Cohort 1 only: Pregnant or breast-feeding females. Female subjects must have a negative pregnancy test at the Part A and Part B Day 1 Visit before receiving the first dose of study drug.
- Cohort 2, Pancreatic Enzyme Replacement Therapy [PERT] sub study only: Meets none of the exclusion criteria for Part A, History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the sub study or pose additional risk to the subject by withdrawing PERT
The study team makes the final eligibility decision.
Where it's taking place
- Germany
- Sweden
- Netherlands
- France
- United Kingdom
- New Zealand
- Australia
- Switzerland
- United States
- Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Germany; Sweden; Netherlands; France; United Kingdom; New Zealand and 4 more locations. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from EU CTIS. The study team decides eligibility, and your own doctor gives medical advice.