None listed
Conditions
Brief summary
In this study we want to look at the impact starting on the new treatment for cystic fibrosis (CF) - Elexacaftor/tezacaftor/ivacaftor (ETI – also known as Trikafta) has on children ages 6-11 years in Australia. In the clinical trials conducted before a drug is approved for sale, there are various controls and measures around which children receive the treatment and what is measured. The results of these clinical trials showed that ETI is likely to improve lung function and health related quality of life. This study is about finding out if the same improved lung function and quality of life is seen in the real world, where there are less controls around which children can take the medication. We are adding this study to the BEAT CF project as the data we need to work out the impact of Trikafta in the real world is the same type of data that is being collected in BEAT CF.
Interventions
Elexacaftor/tezacaftor/ivacaftor (ETI) is a novel therapy for cystic fibrosis (CF) that has recently been introduced into the 6-11 year old age group in Australia. Clinical trials sponsored by the ETI product manufacturer have indicated that ETI is likely to result in improved lung function and health related quality of life. However, there is a knowledge gap regarding the impact of ETI when used in the real world. This study seeks to address this knowledge by assessing the impact of ETI, using
Elexacaftor/tezacaftor/ivacaftor (ETI) is a novel therapy for cystic fibrosis (CF) that has recently been introduced into the 6-11 year old age group in Australia. Clinical trials sponsored by the ETI product manufacturer have indicated that ETI is likely to result in improved lung function and health related quality of life. However, there is a knowledge gap regarding the impact of ETI when used in the real world. This study seeks to address this knowledge by assessing the impact of ETI, using the BEAT CF platform, when this therapy is commenced in children aged 6-11 years old. Participants in this substudy must already be participating in the BEAT CF study (already registered in ACTRN12621000638831). Eligible participants from the BEAT CF study are those aged 6-11 years old, who are commencing ETI therapy. Participating in this substudy involves doing spirometry, having your weight and height measured and your parents filling out a health related quality of life questionnaire (CFQ-R) at the same times as if you were not in this substudy (your regular clinic appointments), as well as having these measurements about an extra 2-3 times. The timing for these measurements are prior to starting ETI therapy, and approximately 14 days, 1 month, 3 months, and 6 months after.
Sponsors
University of Sydney
Eligibility
Sex/Gender
All
Age
6 Years to 11 Years
Healthy volunteers
No
Inclusion criteria
1. Be Enrolled in the BEAT CF PEx Cohort 2. Be Aged 6 to 11 years old 3. Have provided written informed consent (participant or legally responsible guardian) for participation in the ETI Observations substudy 4. Plan to commenceing ETI therapy within the next 6 months
Exclusion criteria
Responsible Clinician deems enrolment is not in the patient’s best interest.
Outcome results
None listed