ELEMENT
Research type
Research Study
Full title
The Effects Of Casein Glycomacropeptide In Metabolic Control And General Health Status In Children With PKU: A Randomised Crossover Trial
IRAS ID
239520
Contact name
Anita MacDonald
Contact email
Sponsor organisation
Vitaflo (International) Ltd
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 11 months, 28 days
Research summary
This is a randomised controlled trial with a crossover design conducted at several study centres, in the UK and Europe, in 4 to 12 years old children with Phenylketonuria (PKU). The study will be performed over two 12-week test periods comparing casein glycomacropeptide (CGMP) based protein substitute with normal L-amino acid protein substitute (AA) with a washout period of 4 weeks inbetween. When CGMP is given as the study product, the phenylalanine (Phe) content of the CGMP will be deducted from the total daily food allowance from food and drink. 20g protein equivalent supplied by the CGMP provides 36 mg Phe. To compensate for the reduction in energy intake by removing this Phe from food/drink, an additional 100 to 200 kcals will be prescribed daily. Blood Phe levels will be analysed over the study as well as the following parameters: gut health, anthropometric measurements, nutritional status, plasma amino acids, renal, inflammation and oxidative stress as well as acceptability and satiety of the product. Blood spots will be performed weekly at home as well as stool sample collection prior to each study visit. Blood collection will be performed at the hospital in each study visit.
We aim to show the different effects of both protein substitutes on metabolic control as well as potential health benefits of using a casein based protein substitute.
Lay summary of study results: 12 participants were enrolled on the study, aged from 4-9 years. 4 were female 8 were male.
There were no withdrawals.7 participants were randomised to received PKU sphere followed by Amino Acids and 5 participants received Amino Acids followed by PKU sphere.
The findings from this study showed:
Metabolic Control
Metabolic control (phenylalanine / tyrosine levels) for all participants were kept within acceptable range during the study.
• Blood spot Phe levels were higher when taking PKU sphere compared to Amino Acids.
• There was no difference in tyrosine levels.Gastrointestinal Tolerance
PKU sphere gave a statistically significant improvement in several GI symptoms for participants.
Participants taking PKU sphere reported an improvement in symptoms such as, stomach pain, heartburn and reflux, wind/bloating and nausea.
Acceptability & Adherence
Both PKU sphere and amino acid-based substitutes were well received by participants, nearly all were able to take their prescribed intakes.
6/12 participants who completed the study wanted to carry on taking PKU sphere.
There were no differences seen in participants’ satiety levels.
Long Term Health
PKU sphere seemed to show a positive trend on quality of life; participants reported improved gut symptoms with less constipation and discomfort when eating.
There were no statistically significant differences in participants’ blood markers for renal, oxidative stress, inflammation or gut health.
Side Effects
Side effects are undesired events that can happen after taking any dietary or medical product.
During the study there were 14 side effects reported. 13 were classed as mild, 1 was moderate.
None were related to the study product.This study showed that PKU sphere was safe to use.
Conclusion
The ELEMENT study is the first multi-centre study of its kind. The results from this study found that:
• PKU sphere kept markers of metabolic control (phenylalanine / tyrosine levels) within acceptable range for all participants.
• PKU sphere was well tolerated by most participants.
• PKU sphere improved gut symptoms for participants when compared against amino acid-based protein substitutes.
• PKU sphere is safe to use for the dietary management of PKU.
• Overall, PKU sphere offered participants an acceptable protein substitute choice with clinically and statistically proven benefits for the dietary management of PKU.REC name
North West - Liverpool Central Research Ethics Committee
REC reference
19/NW/0032
Date of REC Opinion
20 Feb 2019
REC opinion
Further Information Favourable Opinion