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What’s happening in clinical trials: Kaftrio in children
Accessing effective treatments like Kaftrio at a young age could have huge potential benefits for children with CF, including limiting lung damage early and improving daily life.
However, while we know a lot about the benefits of triple combination CFTR modulator therapies like Kaftrio for many adults with CF, more studies are needed before it can be recommended as a treatment in younger age groups. We also have a lot to understand about what these new treatments could mean for children with CF in the long term.
What do we know so far?
Two recent studies looked at Kaftrio in children aged 6 to 11 years. They both found Kaftrio to be safe and effective for this age group, with children who took part seeing improvements in lung function, sweat chloride levels and BMI. Following these studies, Kaftrio was approved as a treatment for children aged 6-11 in the UK in 2022.
A study published earlier this year found similar findings in children aged 2 to 5 years. Kaftrio has now been approved in the US for this age group and we are waiting for news of whether it will be approved in the UK. We will continue to share any updates we have on this.
What about younger children?
A couple of new studies are looking at triple combination treatments in children as young as 12 months.
Children aged 12 to <24 months:
This study is looking at whether Kaftrio is safe and well tolerated in children aged from 12 months to less than 24 months.
See the Trials Tracker listing
Children aged 1 to 11 years:
This study is looking at an adapted version of Kaftrio in children aged 1 to 11. It has completed recruitment for the 6 to 11 years age group and will open for younger age groups shortly.
See the Trials Tracker listing
Understanding the long-term impact for children
Kaftrio has already shown its potential to change the nature of CF treatment. So what does this mean for children with CF in the long term? Studies are already underway to monitor the effects of Kaftrio as a long-term treatment in children who are able to benefit.
We are also supporting the ENHANCE study, which was announced this month. This study hopes to gain an in-depth understanding of how these changes are affecting all children with CF. It will monitor children over a period of five years to look at how CF develops and affects different groups of children from a young age.
What about children with rare variants?
Our top research priorities include looking at options for people unable to benefit from current modulators. There is a lot of work behind the scenes to address this through the research we support.
The CF BioResource Project plays a key role in this. The aim of this study is to create a register of people with CF based on their genetic make up, to make it easier for researchers to find volunteers and develop studies for the rare forms of CF. Babies, children and adults of any age and gene variant are invited to take part by providing a blood sample. You can find this and other trials currently recruiting or recently closed for recruitment on our Trials Tracker. You can filter by age, location and type of therapy.
Can my child join a trial?
Our Trials Tracker lists all trials currently open to participants in the UK and includes details on who is eligible to take part. Talk to your CF team if you would like more information about trials that are open for your child.
You might find it useful to read our information about talking to children about clinical trials, as well as our Comic Book for children.
Find out more about cystic fibrosis clinical trials and the Clinical Trials Accelerator Platform. You can also find a list of current trials on the Trials Tracker.
It's thanks to our incredible supporters that we can continue to be at the cutting edge of CF research. Making breakthroughs and discoveries that change lives for the better. Now and in the future. But we know this is just the start. Incredible progress has been made, but there is still a long way to go until everyone with CF can truly live a life unlimited. We won’t stop until we can all lead the lives we want. Until cystic fibrosis stops challenging, damaging or shortening lives. And you can be part of it.
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