CHAFfinch Study - Children’s Acceptability of Furosemide
Research type
Research Study
Full title
Acceptability of age-appropriate furosemide orodispersible tablets (ODTs) in the paediatric population
IRAS ID
339343
Contact name
Daniel Hawcutt
Contact email
Duration of Study in the UK
1 years, 0 months, 0 days
Research summary
Research Summary: In the UK, medicines are carefully regulated to ensure that they are as safe as possible, work as intended and are made of suitable quality. Medicines are granted a product licence, now known as a marketing authorisation (MA), by the Medicines and Healthcare products Regulatory Agency (MHRA) and the European Medicines Agency (EMA) when they are deemed to have met these strict safety and quality standards. As part of these regulations, manufacturers must include an assessment of how acceptable the medicine is for the intended user, before it is made available on the market, for wider use.
Proveca Ltd (https://www.proveca.com/) is a pharmaceutical company, which specialises in developing medicines for children. The Paediatric Medicines Research Unit (PMRU) Team (https://alderhey.nhs.uk/healthier-future/research/paediatric-medicines-research-unit) are working with Proveca Ltd to help improve medicines available for Children and Young People (CYP) by tailoring any future medicines developed to the specific needs of children.
Historically, medicines have not always been designed with children in mind. Many medicines are only available in dosage forms, which are more suitable for use in adults. This leads to medicines having to be used in untested ways (so called “off-label” (OL) use), as they may not have been tested for use in certain age groups or they may need to be adapted / modified (e.g. a tablet is crushed and mixed with water) to allow administration to a child. Alternatively, unlicensed (UL) medicines may need to be sourced and purchased to fulfil this unmet clinical need.
One of the medicines identified which could benefit from an improved formulation for use in children is furosemide. A new orodispersible tablet (ODT) formulation of furosemide has been developed by Proveca Ltd which is specifically designed for use in children. Furosemide is a diuretic. This makes your body get rid of any excess bodily fluid (oedema) through your kidneys. It can be used to treat different conditions such as high blood pressure, heart failure or help when your kidneys are not working properly.
The aim of the study is to investigate, within a small group of patients who are already prescribed and taking oral furosemide, how they react to the new formulation. How acceptable is it to them? Do they like it? Do the results suggest that the product would be acceptable to a wider paediatric population?
This acceptability study involves a one-off, direct switch of the furosemide formulation being administered. The participant will be asked to take the same dose of furosemide (known as their current ‘regular dose’) using the new ODT formulation provided. This will happen for one dose only and the researcher will observe them taking the new formulation and complete a short feedback questionnaire. Parents and participants (if willing/able) will have the option to provide additional feedback via an appropriate questionnaire. There will be no further follow-up.
Summary of Results: This study assessed the acceptability of a new furosemide orodispersible tablet (ODT) for children aged from birth up to 12 years old.
Rather than taking their usual form of furosemide, such as an oral liquid, the children were given the furosemide ODT, at the same dose. Ten children took part in the study, including three babies who were younger than 2 months old. Half of the participants were boys. Participant ages ranged from 28 days to 6 years. Most had been taking furosemide for less than three months.
The doses ranged from 2mg to 20mg, often requiring multiple tablets to be given, using the 1mg or 5mg ODTs. The ODT was placed in the child’s cheek or on the tongue to dissolve. They were administered either by a parent, a researcher, or, in one instance, by the child. Most children also had a drink of water or milk to help to dissolve the tablet.
All ten children successfully took the full dose of furosemide. As a result, no replacement doses using their regular medication were necessary.
Researchers also checked how pleasant the tablets were to take. They did this by watching the videos of the children taking the tablets and noting their facial expressions and reactions. Six children seemed to find it ‘unpleasant’, whilst four had a ‘neutral’ reaction. The study didn’t compare how pleasant the children found the new ODT compared to their regular furosemide medicine.
To measure how acceptable the tablet was overall, researchers used a standard scoring tool (known as the composite endpoint). They found that for four children, the acceptability of the tablet was good. For the other six, it was rated as low. Despite this, none of the children refused to take it, and both parents and children gave positive feedback. Recording the time taken for the ODT to dissolve in the mouth was not part of the study. However, it was noted that the ODT took longer to dissolve in the children below 2 months of age compared to those that were older.
Parents also filled out a study questionnaire. 90% of parents said the tablet was acceptable for their child and would be willing to give it again. Two children, who were old enough to give their opinion, also said they would be happy to take it again.
REC name
West Midlands - South Birmingham Research Ethics Committee
REC reference
24/WM/0055
Date of REC Opinion
15 Apr 2024
REC opinion
Further Information Favourable Opinion