A Phase 3 Open-label Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older Homozygous or Heterozygous for the F508del-CFTR Mutation

Study on Safety and Tolerability of Investigational Medication for Genetic Disorder Affecting Lungs and Digestion

Recruiting
6 years - 100 years
All
Phase N/A
1 Location

Brief description of study.

The purpose of this study is to evaluate the long-term safety and tolerability of TEZ/IVA in subjects with cystic fibrosis aged 6 years and older, who are homozygous or heterozygous for F508del mutation.

Detailed description of study

The purpose of this study is to evaluate the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in subjects with cystic fibrosis (CF) aged 6 years and older, homozygous or heterozygous for the F508del mutation.
Participants will not be paid for their participation.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: cystic fibrosis
  • Age: 6 years - 100 years
  • Gender: All

Inclusion Criteria
Male and female subjects with CF aged 6 years and older, who completed Study 113
Part B or Study 115 and are
homozygous for F508del (F/F),
heterozygous for F508del and a second allele with a residual CFTR-function
mutation (F/RF), or
heterozygous for F508del and a second allele that has been shown to be
responsive to TEZ/IVA
Exclusion Criteria
History of any comorbidity 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
Pregnant and nursing females
Sexually active subjects of reproductive potential who are not willing to follow the
contraception requirements
History of drug intolerance in the parent study that would pose an additional risk to the
ubject in the opinion of the investigator, and which should be discussed with the Vertex
medical monitor
History of poor compliance with study drug and/or procedures in a previous study as deemed
by the investigator
Ongoing participation in another study with investigational drug

Updated on 19 Feb 2024. Study ID: 1802366632

This study investigates the safety and tolerability of an investigational medication for cystic fibrosis. Cystic fibrosis is a genetic disorder that affects the lungs and digestive system. The study focuses on individuals aged 6 years and older who have a specific genetic mutation called F508del.

Participants will either receive the investigational medication or a placebo, which is an inactive substance that looks like the investigational medication but does not contain any medicine. The study will monitor participants for any side effects and how well they tolerate the treatment.

  • Who can participate: Participants must be 6 years or older with cystic fibrosis and have the F508del mutation in both gene copies or in combination with another responsive mutation.
  • Study details: Participants will receive either the investigational medication or a placebo. A placebo is an inactive substance that looks like the investigational medication but does not contain any medicine.

Find a site

Message sent successfully.
We have submitted the information you provided to the research team at the location you chose. For your records, we have sent a copy of the message to your email address.
If you would like to be informed of other studies that may be of interest to you, you may sign up for Patient Notification Service.
Sign up

Send a message

Enter your contact details to connect with study team

Investigator Avatar

Primary Contact

First name*
Last name*
Email*
Phone number*
Preferred way of contact label
Race
Ethnicity
Other language

Default Content Message Here