REVEAL: PaRents pErspectiVEs of KAftrio in chiLdren aged 2-5
Research type
Research Study
Full title
REVEAL: A Multi-disciplinary team facilitated parental/ guardian focus group exploring perceptions, expectations and realities of Kaftrio in children with Cystic Fibrosis aged 2-5.
IRAS ID
329535
Contact name
Kelly Davies
Contact email
Sponsor organisation
Alder Hey NHS foundation Trust
Clinicaltrials.gov Identifier
7/8/23, SOC approval
Duration of Study in the UK
1 years, 10 months, 16 days
Research summary
Cystic fibrosis (CF) is a genetic condition that causes thick sticky respiratory secretions that leads to a vicious cycle of recurrent chest infections, inflammation and airway damage resulting in premature death. In recent years highly effective modulator therapy represents a significant step forward in CF care and is expected to have a significant impact on the health and mortality of many individuals with CF. In March 2020 Kaftrio became available for children aged 12+, the licencing for this then dropped to children aged 6 to 12 in January of 2022. Since then clinical teams have reported improvement in lung function and reduced rates of exacerbations and use of antibiotics. Some individuals have also reported medication side effects and psychological impact. It is expected that as soon as Autumn/Winter 2023, Kaftrio will be available for children aged between 2-6 years of age. It is important clinical teams understand the perceptive and experiences of families prior to commencing this as they navigate starting a new medication.
REC name
London - Surrey Research Ethics Committee
REC reference
23/PR/0998
Date of REC Opinion
22 Sep 2023
REC opinion
Further Information Favourable Opinion