Clinical Trial Details
— Status: Completed
Administrative data
NCT number |
NCT05062226 |
Other study ID # |
GMP2020 |
Secondary ID |
|
Status |
Completed |
Phase |
N/A
|
First received |
|
Last updated |
|
Start date |
September 16, 2020 |
Est. completion date |
November 24, 2023 |
Study information
Verified date |
November 2023 |
Source |
Nutricia UK Ltd |
Contact |
n/a |
Is FDA regulated |
No |
Health authority |
|
Study type |
Interventional
|
Clinical Trial Summary
Three new protein substitutes have been developed to help with compliance in PKU and TYR
patients, which are powdered protein substitutes, low in Phe and, low in Phe and Tyr
respectively, with a mixed balance of glycomacropeptide (GMP), essential and non-essential
amino acids, carbohydrates, fats and micronutrients for the dietary management of PKU and
TYR.
This series of case-studies will evaluate the acceptability, compliance, gastrointestinal
tolerance and safety of the three GMP-based products for PKU and TYR in 45 patients with PKU
and TYR, in both adults and children over 3 years (15 patients per product). The case study
will last 29 days in total, including a 1-day baseline period followed by a 28-day
intervention period. The case studies will be conducted across multiple specialist metabolic
centres in the UK, to meet the UK ACBS and GMS requirements for acceptability studies. A
series of case studies is undertaken due to the rarity of these conditions and the difficulty
in recruiting these patients to trials.
Description:
Phenylketonuria (PKU) and Tyrosinaemia (TYR) are rare inherited metabolic disorders of amino
acid metabolism requiring dietary management. PKU is characterised by an inability to
metabolise the essential dietary amino acid phenylalanine (Phe) into tyrosine (Tyr). As such,
without appropriate management, PKU can lead to extremely elevated levels of Phe within the
blood and brain, which can be detrimental to neurological health. The dietary management of
PKU involves restricting dietary sources of Phe. Tyrosinaemia (TYR) is characterised by the
inability to metabolise the dietary amino acid Tyr. If left uncontrolled, TYR can lead to
serious hepatic, renal and neurological complications. The dietary management of TYR involves
restriction of dietary sources of Tyr and Phe (as Phe is converted into Tyr). The dietary
management of PKU and TYR involves restricting dietary protein intake to promote optimal
metabolic control, whilst meeting all other amino acid and nutrient requirements. As Phe and
Tyr are present in significant quantities in nearly all dietary proteins, this usually
requires a very low protein diet from food, supplemented with a Phe-free (for PKU) or Phe-
and Tyr-free (for TYR) amino acid-based protein substitute. However, compliance with the diet
is often poor, with patients commonly going "off diet" during adolescence and in adulthood.
This can lead to entirely preventable, lifelong mental and physiological impairments. Reasons
for non-compliance often revolve around the strong taste of amino acid-based protein
substitutes, alongside the inability to conform to the normal eating habits of peers.
Three new protein substitutes have been developed to help with compliance in PKU and TYR
patients, which are powdered protein substitutes, low in Phe and, low in Phe and Tyr
respectively, with a mixed balance of glycomacropeptide (GMP), essential and non-essential
amino acids, carbohydrates, fats and micronutrients for the dietary management of PKU and
TYR. The protein in GMP-based products for PKU and TYR are based on GMP, a peptide isolated
from milk during cheese manufacturing and the only known naturally derived protein source low
in Phe and Tyr. The GMP is supplemented with appropriate quantities of other amino acids
which are naturally low or not present in GMP, as well as carbohydrates and fats. This
differs from traditional formulae which are based wholly on individual, synthetically
produced amino acids to provide the source of dietary nitrogen, supplemented with vitamins
and minerals. Studies to date have illustrated that PKU patients who replace their regular
Phe-free amino acid-based formula with GMP-based foods may have better diet compliance,
prefer the taste and may have improved blood Phe control.
This series of case-studies will evaluate the acceptability, compliance, gastrointestinal
tolerance and safety of the three GMP-based products for PKU and TYR in 45 patients with PKU
and TYR, in both adults and children over 3 years (15 patients per product). The case study
will last 29 days in total, including a 1-day baseline period followed by a 28-day
intervention period. The case studies will be conducted across multiple specialist metabolic
centres in the UK, to meet the UK ACBS and GMS requirements for acceptability studies. A
series of case studies is undertaken due to the rarity of these conditions and the difficulty
in recruiting these patients to trials.