Back to resultsThe Effects of CGMP in Children and Adults With PKU (ELEMENT)
Primary Purpose
Phenylketonurias
Status
Completed
Phase
Not Applicable
Locations
International
Study Type
Interventional
Intervention
CGMP protein substitute
L-amino acid protein substitute
Sponsored by
About this trial
This is an interventional treatment trial for Phenylketonurias focused on measuring Phenylketonuria, PKU, Child, Adult, GMP, Glycomacropeptide
Eligibility Criteria
Inclusion Criteria:
- PKU patient diagnosed on newborn screening (NBS).
- > 50% of protein intake from protein substitutes.
- (CHILDREN) Dietary intake of ≤ 1000 mg Phe from natural protein or ≤20 g natural protein inclusive of fruit and vegetables per day.
- (ADULTS) Dietary intake of ≤ 1500 mg Phe from natural protein or ≤ 30 g natural protein inclusive of fruit and vegetables per day.
- (CHILDREN) Three out of the last four consecutive Phe measurements within the target range (≤360 µmol/L in patients aged 4-12 years).
- (ADULTS) Two out of the last four consecutive Phe measurements within the target range (≤600 µmol/L for adults).
- Male or female aged 4-12 years or 18 years and over.
- Early and continuously treated. Adherent to their prescribed PKU diet consisting of a protein-restricted diet and free AA based protein substitute.
- Otherwise in good general health as evidenced by medical history.
- Able to provide written informed consent (patient or parent/guardian).
- Able to comply with the study protocol and take study product according to the opinion of the PI.
- Protein substitute intake provided by L-amino acid supplements only.
- (ADULTS) No studies have been done in pregnant women. To ensure patients safety, female patients of childbearing potential must have a negative pregnancy test prior to completing the screening procedures.
- (ADULTS) All female patients of childbearing potential and sexually mature males should be willing to use a medically accepted method of contraception throughout the study.
- Successful 3-day PKU Sphere taste test.
Exclusion Criteria:
- Concomitant diseases / disorders such as but not limited to renal or gut disease / disorders and diabetes.
- Currently or previously treated with tetrahydrobiopterin (BH4), pegylated recombinant phenylalanine ammonia lyase (PEG PAL), large neutral amino acids.
- Previous intake of CGMP for more than four consecutive weeks.
- Having a current infection.
- Known soya, milk or fish allergies / intolerance.
- Patients who are currently participating in, plan to participate in, or have participated in an interventional investigational drug, food or medical device trial within 30 days prior to screening visit.
- Where applicable, patients not covered by Health Insurance System and/or not in compliance with the recommendations of National Law in force.
Sites / Locations
- Rigshospitalet
- Bristol Royal Hospital for Children
- Birmingham Children's Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Experimental
Arm Label
L-amino Acids
PKU Sphere
Arm Description
The specific amount and timing of L-amino acid consumption will be at the instruction of a dietitian following a review of the individual patient's dietary needs. Consumption period: 12 weeks.
The specific amount and timing of PKU sphere consumption will be at the instruction of a dietitian following a review of the individual patient's dietary needs. Consumption period: 12 weeks.
Outcomes
Primary Outcome Measures
Change in overnight fasting plasma Phe
Overnight fasting plasma phenylalanine level (μmol/l)
Change in overnight fasting plasma Tyr
Overnight fasting plasma tyrosine level (μmol/l)
Change in overnight fasting blood Phe
Overnight fasting blood phenylalanine level (μmol/l) using blood spots
Change in overnight fasting blood Tyr
Overnight fasting blood tyrosine level (μmol/l) using blood spots
Secondary Outcome Measures
Change in gut health: PedsQL Gastrointestinal Symptoms Scales, Version 3.0
Five point frequency scale for each question: Never (0), Almost Never (1), Sometimes (2), Often (3), Almost Always (4).
Change in gut health: Stool pH for reducing sugars
From stool sample. To rule out carbohydrate intolerance as a cause of any irritable bowel syndrome symptoms
Change in gut health: Stool calprotectin
From stool sample
Change in gut health: Short chain fatty acids
From stool sample
Change in gut health: Immunoglobulin A
From stool sample
Change in gut health: Bristol stool form scale
Bristol stool form scale:
Type 1: Separate hard lumps, like nuts (hard to pass)
Type 2: Sausage-shaped, but lumpy
Type 3: Like a sausage but with cracks on its surface
Type 4: Like a sausage or snake, smooth and soft
Type 5: Soft blobs with clear cut edges (easy to pass)
Type 6: Fluffy pieces with ragged edges, a mushy stool
Type 7: Watery, no solid pieces, entirely liquid
Change in gut health: 3 day weighed diet diary
3 day weighed diet diary
Change in anthropometric measurements: weight
Weight (kg)
Change in anthropometric measurements: height
Height (cm)
Change in Albumin
Following overnight fasting
Change in Pre-albumin
Following overnight fasting
Change in Transferrin
Following overnight fasting
Change in Retinol-binding protein
Following overnight fasting
Change in Glucose
Following overnight fasting
Change in Haemoglobin A1C
Following overnight fasting
Change in IGF-1
Following overnight fasting
Change in C-peptide
Following overnight fasting
Change in Short chain fatty acids
Following overnight fasting
Change insulin production
Following overnight fasting
Change in alanine
Total plasma amino acids following overnight fasting
Change in arginine
Total plasma amino acids following overnight fasting
Change in asparagine
Total plasma amino acids following overnight fasting
Change in citrulline
Total plasma amino acids following overnight fasting
Change in cystine
Total plasma amino acids following overnight fasting
Change in glutamate
Total plasma amino acids following overnight fasting
Change in glutamine
Total plasma amino acids following overnight fasting
Change in glycine
Total plasma amino acids following overnight fasting
Change in histidine
Total plasma amino acids following overnight fasting
Change in isoleucine
Total plasma amino acids following overnight fasting
Change in leucine
Total plasma amino acids following overnight fasting
Change in lysine
Total plasma amino acids following overnight fasting
Change in methionine
Total plasma amino acids following overnight fasting
Change in proline
Total plasma amino acids following overnight fasting
Change in ornithine
Total plasma amino acids following overnight fasting
Change in phenylalanine
Total plasma amino acids following overnight fasting
Change in serine
Total plasma amino acids following overnight fasting
Change in taurine
Total plasma amino acids following overnight fasting
Change in threonine
Total plasma amino acids following overnight fasting
Change in tryptophan
Total plasma amino acids following overnight fasting
Change in tyrosine
Total plasma amino acids following overnight fasting
Change in valine
Total plasma amino acids following overnight fasting
Change in renal biomarkers: Serum cystatin C
Blood sample following overnight fasting
Change in renal biomarkers: Serum creatinine
Blood sample following overnight fasting
Change in renal biomarkers: Blood urea nitrogen
Blood sample following overnight fasting
Change in Adiponectin
Following overnight fasting. An anti-inflammatory marker and related with both adipose tissue and insulin sensitivity
Change in C-reactive protein (high sensitivity measurement)
Following overnight fasting
Change in Serum myeloid-related protein
Following overnight fasting
Change in Soluble Interleukin 2 Receptor
Following overnight fasting
Change in Inflammatory cytokine panel
Following overnight fasting
Change in oxidative stress: White cells glutathione (GSH)
Following overnight fasting
Change in oxidative stress: Plasma thiobarbituric acid-reactive species (TBAR)
Following overnight fasting
Change in oxidative stress:Total antioxidant reactivity (TAR)
Following overnight fasting
Change in patient opinion of acceptability of the product
Product acceptability Likert scale:
I loved it (5 - best)
I liked it (4)
I neither liked nor disliked it (3)
I didn't like it (2)
I really didn't like it (1 - worst)
Change in hunger
Measuring hunger in primary school children Likert scale:
I am really hungry! My belly feels very empty and is rumbling! (1 - worst)
I am quite hungry and my belly feels a little empty. (2)
I feel just right, not too hungry and not too full. (3)
I am quite full, but there is still a little room in my belly. (4)
I am not hungry at all! My belly feels very full and I cannot eat any more food! (5 - best)
Full Information
NCT ID
NCT04076176
First Posted
May 30, 2019
Last Updated
August 18, 2023
Sponsor
Vitaflo International, Ltd
Collaborators
Arla Foods
1. Study Identification
Unique Protocol Identification Number
NCT04076176
Brief Title
The Effects of CGMP in Children and Adults With PKU
Acronym
ELEMENT
Official Title
The Effects Of Casein Glycomacropeptide On Metabolic Control And General Health Status In Children And Adults With PKU: A Randomised Crossover Trial
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Completed
Study Start Date
April 26, 2019 (Actual)
Primary Completion Date
January 31, 2023 (Actual)
Study Completion Date
January 31, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vitaflo International, Ltd
Collaborators
Arla Foods
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Randomised controlled trial with a crossover design. For early and continuously treated patients with phenylketonuria (PKU) that are adherent. Two 12-week periods where patients consume either casein glycomacropeptide (CGMP) based protein substitute or a free amino acid (AA) based protein substitute. 4 week wash out period in between. The protein substitutes will be consumed daily together with the patient's regular low protein diet.
Detailed Description
The study is a randomised controlled trial with a crossover design conducted at several international study centres.
The study population is early and continuously treated patients with PKU that are adhering to their prescribed diet.
The participants will be given both a CGMP based protein substitute and a free AA based protein substitute as their main protein source on two different 12-week periods in a crossover design. The two periods will be separated by a 4 week wash out period.
The protein substitutes will be consumed daily together with the patient's regular low protein diet during the intervention periods.
Primary Objective The primary objective of the study is to investigate the effects of a CGMP based formula compared to an AA based formula upon mean plasma Phe levels after 12 weeks of daily intake in patients with PKU.
Secondary Objectives The secondary objectives of the study are to investigate if a CGMP based formula compared to an AA based formula in PKU patients provides any long-term health benefits concerning gut health, inflammation, oxidative stress and product acceptability.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Phenylketonurias
Keywords
Phenylketonuria, PKU, Child, Adult, GMP, Glycomacropeptide
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Crossover Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
12 (Actual)
8. Arms, Groups, and Interventions
Arm Title
L-amino Acids
Arm Type
Active Comparator
Arm Description
The specific amount and timing of L-amino acid consumption will be at the instruction of a dietitian following a review of the individual patient's dietary needs.
Consumption period: 12 weeks.
Arm Title
PKU Sphere
Arm Type
Experimental
Arm Description
The specific amount and timing of PKU sphere consumption will be at the instruction of a dietitian following a review of the individual patient's dietary needs.
Consumption period: 12 weeks.
Intervention Type
Dietary Supplement
Intervention Name(s)
CGMP protein substitute
Other Intervention Name(s)
PKU Sphere
Intervention Description
PKU sphere is a powdered, low phenylalanine protein substitute, containing a balanced mix of casein glycomacropeptide (CGMP) isolate, essential and non-essential amino acids, carbohydrate, vitamins, minerals, trace elements and the omega-3 long chain polyunsaturated fatty acid (LCP); docosahexaenoic acid.
Intervention Type
Dietary Supplement
Intervention Name(s)
L-amino acid protein substitute
Intervention Description
Patient may choose from a list of products, specified in the protocol, that are nutritionally comparable to PKU Sphere.
Primary Outcome Measure Information:
Title
Change in overnight fasting plasma Phe
Description
Overnight fasting plasma phenylalanine level (μmol/l)
Time Frame
Measured at days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5)
Title
Change in overnight fasting plasma Tyr
Description
Overnight fasting plasma tyrosine level (μmol/l)
Time Frame
Measured at days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5)
Title
Change in overnight fasting blood Phe
Description
Overnight fasting blood phenylalanine level (μmol/l) using blood spots
Time Frame
Measured twice per week up to 32 weeks
Title
Change in overnight fasting blood Tyr
Description
Overnight fasting blood tyrosine level (μmol/l) using blood spots
Time Frame
Measured twice per week up to 32 weeks
Secondary Outcome Measure Information:
Title
Change in gut health: PedsQL Gastrointestinal Symptoms Scales, Version 3.0
Description
Five point frequency scale for each question: Never (0), Almost Never (1), Sometimes (2), Often (3), Almost Always (4).
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in gut health: Stool pH for reducing sugars
Description
From stool sample. To rule out carbohydrate intolerance as a cause of any irritable bowel syndrome symptoms
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in gut health: Stool calprotectin
Description
From stool sample
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in gut health: Short chain fatty acids
Description
From stool sample
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in gut health: Immunoglobulin A
Description
From stool sample
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in gut health: Bristol stool form scale
Description
Bristol stool form scale:
Type 1: Separate hard lumps, like nuts (hard to pass)
Type 2: Sausage-shaped, but lumpy
Type 3: Like a sausage but with cracks on its surface
Type 4: Like a sausage or snake, smooth and soft
Type 5: Soft blobs with clear cut edges (easy to pass)
Type 6: Fluffy pieces with ragged edges, a mushy stool
Type 7: Watery, no solid pieces, entirely liquid
Time Frame
Completed for 7 days prior to days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in gut health: 3 day weighed diet diary
Description
3 day weighed diet diary
Time Frame
Completed for 3 days prior to days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in anthropometric measurements: weight
Description
Weight (kg)
Time Frame
Days -31 (visit 1), 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in anthropometric measurements: height
Description
Height (cm)
Time Frame
Days -31 (visit 1), 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in Albumin
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in Pre-albumin
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in Transferrin
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in Retinol-binding protein
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in Glucose
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in Haemoglobin A1C
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in IGF-1
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in C-peptide
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in Short chain fatty acids
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change insulin production
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in alanine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in arginine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in asparagine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in citrulline
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in cystine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in glutamate
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in glutamine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in glycine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in histidine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in isoleucine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in leucine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in lysine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in methionine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in proline
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in ornithine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in phenylalanine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in serine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in taurine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in threonine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in tryptophan
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in tyrosine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in valine
Description
Total plasma amino acids following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in renal biomarkers: Serum cystatin C
Description
Blood sample following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in renal biomarkers: Serum creatinine
Description
Blood sample following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in renal biomarkers: Blood urea nitrogen
Description
Blood sample following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in Adiponectin
Description
Following overnight fasting. An anti-inflammatory marker and related with both adipose tissue and insulin sensitivity
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in C-reactive protein (high sensitivity measurement)
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in Serum myeloid-related protein
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in Soluble Interleukin 2 Receptor
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in Inflammatory cytokine panel
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in oxidative stress: White cells glutathione (GSH)
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in oxidative stress: Plasma thiobarbituric acid-reactive species (TBAR)
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in oxidative stress:Total antioxidant reactivity (TAR)
Description
Following overnight fasting
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5).
Title
Change in patient opinion of acceptability of the product
Description
Product acceptability Likert scale:
I loved it (5 - best)
I liked it (4)
I neither liked nor disliked it (3)
I didn't like it (2)
I really didn't like it (1 - worst)
Time Frame
Days 84 (visit 3) and 196 (visit 5)
Title
Change in hunger
Description
Measuring hunger in primary school children Likert scale:
I am really hungry! My belly feels very empty and is rumbling! (1 - worst)
I am quite hungry and my belly feels a little empty. (2)
I feel just right, not too hungry and not too full. (3)
I am quite full, but there is still a little room in my belly. (4)
I am not hungry at all! My belly feels very full and I cannot eat any more food! (5 - best)
Time Frame
Days 0 (visit 2), 84 (visit 3), 112 (visit 4) and 196 (visit 5)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
PKU patient diagnosed on newborn screening (NBS).
> 50% of protein intake from protein substitutes.
(CHILDREN) Dietary intake of ≤ 1000 mg Phe from natural protein or ≤20 g natural protein inclusive of fruit and vegetables per day.
(ADULTS) Dietary intake of ≤ 1500 mg Phe from natural protein or ≤ 30 g natural protein inclusive of fruit and vegetables per day.
(CHILDREN) Three out of the last four consecutive Phe measurements within the target range (≤360 µmol/L in patients aged 4-12 years).
(ADULTS) Two out of the last four consecutive Phe measurements within the target range (≤600 µmol/L for adults).
Male or female aged 4-12 years or 18 years and over.
Early and continuously treated. Adherent to their prescribed PKU diet consisting of a protein-restricted diet and free AA based protein substitute.
Otherwise in good general health as evidenced by medical history.
Able to provide written informed consent (patient or parent/guardian).
Able to comply with the study protocol and take study product according to the opinion of the PI.
Protein substitute intake provided by L-amino acid supplements only.
(ADULTS) No studies have been done in pregnant women. To ensure patients safety, female patients of childbearing potential must have a negative pregnancy test prior to completing the screening procedures.
(ADULTS) All female patients of childbearing potential and sexually mature males should be willing to use a medically accepted method of contraception throughout the study.
Successful 3-day PKU Sphere taste test.
Exclusion Criteria:
Concomitant diseases / disorders such as but not limited to renal or gut disease / disorders and diabetes.
Currently or previously treated with tetrahydrobiopterin (BH4), pegylated recombinant phenylalanine ammonia lyase (PEG PAL), large neutral amino acids.
Previous intake of CGMP for more than four consecutive weeks.
Having a current infection.
Known soya, milk or fish allergies / intolerance.
Patients who are currently participating in, plan to participate in, or have participated in an interventional investigational drug, food or medical device trial within 30 days prior to screening visit.
Where applicable, patients not covered by Health Insurance System and/or not in compliance with the recommendations of National Law in force.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Anita MacDonald
Organizational Affiliation
Birmingham Women's and Children's NHS Foundation Trust
Official's Role
Principal Investigator
Facility Information:
Facility Name
Rigshospitalet
City
Kobenhavn
ZIP/Postal Code
2100
Country
Denmark
Facility Name
Bristol Royal Hospital for Children
City
Bristol
State/Province
Avon
ZIP/Postal Code
BS2 8BJ
Country
United Kingdom
Facility Name
Birmingham Children's Hospital
City
Birmingham
State/Province
West Midlands
ZIP/Postal Code
B4 6NH
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
The Effects of CGMP in Children and Adults With PKU
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