Status:
COMPLETED
The Effects of CGMP in Children and Adults With PKU
Lead Sponsor:
Vitaflo International, Ltd
Collaborating Sponsors:
Arla Foods
Conditions:
Phenylketonurias
Eligibility:
All Genders
4+ years
Phase:
NA
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 ...
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 ...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
April 26 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 31 2023
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT04076176
Start Date
April 26 2019
End Date
January 31 2023
Last Update
August 20 2025
Active Locations (3)
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1
Rigshospitalet
Copenhagen, Denmark, 2100
2
Bristol Royal Hospital for Children
Bristol, Avon, United Kingdom, BS2 8BJ
3
Birmingham Children's Hospital
Birmingham, West Midlands, United Kingdom, B4 6NH