A study of the combined effects of three CFTR modulator medicines in children with cystic fibrosis aged 6 to 11 (VX18-445-106)

Details

Therapeutic approach: Restore CFTR Function
Trial status: Closed with results Participating Centres
Trials Tracker ID: TT002081
Last updated: 13/11/2020

Full title

A Phase 3 study of the efficacy, safety and the body’s effects on a triple combination therapy of VX-445, tezacaftor and ivacaftor in children with cystic fibrosis aged 6 to 11

Study details

VX-445 is an investigational medicine being studied to see how well it works and how safe it is in children with cystic fibrosis aged 6 to 11 years, when taken in combination with tezacaftor and ivacaftor. All three medicines are CFTR modulators which means that they help the faulty CFTR protein found in cystic fibrosis to work properly. Tezacaftor is already approved for use and is what is known as a ‘corrector’. Ivacaftor is also already approved for use and is what is known as a ‘potentiator’. VX-445 is a corrector

Study results

This study looked at the safety and effectiveness of ELX/TEZ/IVA treatment (also known as Kaftrio) in children aged 6 to 11 who have at least one F508del gene variant.

66 children took part in the study and were given Kaftrio. They saw a 10% improvement in lung function on average and improvements in lung-related quality of life. Their sweat chloride levels decreased, which suggests their CFTR function improved. Their BMI also increased, and this was a bigger improvement than seen with other modulator treatments.

These results are consistent with previous studies in older age groups that show this treatment is safe and effective for people who have at least one F508del gene variant. It seemed to be more effective than existing modulator treatments. This study adds to the evidence supporting the use of Kaftrio in this age group.

Link to full results https://clinicaltrials.gov/study/NCT03691779

Phase The different stages involved in the development of a new medication. Phase I focuses on initial safety in people. Phase 2 evaluates safety, correct dose and early signs of whether the medication works. Phase 3 is the stage before medication licensing and looks at safety and medication effectiveness. Phase 4 evaluates longer term use of a medication after it has been licensed for use: Phase 3
CF sponsor: Vertex Pharmaceuticals Incorporated
CF sponsor type: Commercial

CF centres running this trial

Closed

Birmingham Children's Hospital

CTAP centre

NHS Trust: Birmingham Women's and Children's NHS Foundation Trust
Address: Steelhouse Lane
B4 6NH
Local site investigator: Maya Desai
Trial Coordinators: Gurcharan (Robbie) Singh
[email protected]