Evaluation of Low Phenylalanine Formulas

Phenylketonurias Clinical Trial

Official title:

The Effects of an Innovative Glycomacropeptide Based Protein Supplement Purified to Lower the Phenylalanine Content for the Treatment of Paediatric Patients With Phenylketonuria

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT06332105
• Other study ID #: CUK001
• Status: Active, not recruiting
• Phase: N/A
• Start date: January 30, 2023
• Est. completion date: April 2025

Study information

• Verified date: April 2024
• Source: Ajinomoto Co., Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Ajinomoto Cambrooke has developed a PKU protein substitute that is a proprietary blend of purified Glycomacropeptide (GMP) and essential amino acids, under the brand name Glytactin®. One serving of such Glytactin® products contains 20mg or less of Phenylalanine (Phe). The aim of the proposed study is to use this purified GMP-AA-based protein substitute, with less Phe per gram of protein equivalent than other commercially available products, in children with PKU at 100% of their protein substitute intake and evaluate its efficacy and the change in blood Phe in comparison to Phe-free L-AA-based protein substitutes.

Description:

Study Design This is a 2-stage, 15-week randomized crossover trial. The randomized crossover design was chosen as it reduces the influence of differences among individuals; and it offers statistical efficiency (requires fewer subjects than non-crossover designs).

In summary, 2 groups of 9 participants each will be assigned to either (1) sequence 1: take GMP-AA-based PS for the first 4 weeks and then take only L-AA-based Protein Substitute for 4 weeks, or (2) take only L-AA-based Protein Substitute for the first 4 weeks and then take GMP-AA-based PS for 4 weeks. At the end of the first 4 weeks, a 2-week washout period will follow with both groups only consuming L-AA-based PS. Randomization will be generated by a block randomization system and the random order will be kept within a sealed envelope.

Primary research objective The principal research objective of this study is to evaluate the effect on Phe levels of a low-Phe diet combined with a purified GMP-AA-based protein substitute (containing 1 mg Phe/g Protein Equivalent), in the treatment of paediatric patients with PKU.

Secondary research objectives The secondary objectives of the study aim to investigate whether there are any differences between the GMP-AA-based protein substitute and L-AA-based protein Substitute in the frequency and quantity of protein substitute intake, if any GI (gastrointestinal) symptoms occur with ingestion of the GMP-AA-based protein substitute, effect on satiety (hunger) and mood and any differences in anthropometric data.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 19
• Est. completion date: April 2025
• Est. primary completion date: January 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 16 Years

Eligibility

Inclusion Criteria:

• Patients with PKU, aged 5 to 16 years of age, currently compliant with protein restricted diet and L-AA- and/or GMP-AA-based protein substitute willing to switch to a GMP-AA-based only product for 4 weeks.

• 2 out of 4 last blood Phe levels within target range (i.e. 50%): Target range 120-360µmol/l <12 years Target range 120-600µmol/l >12 years

Exclusion Criteria:

• • Milk protein allergy

• Pregnancy

• Severe medical diagnosis not related to PKU

• Treatment with Sapropterin hydrochloride (KUVAN)

Related Conditions & MeSH terms

• Phenylketonurias

Intervention

Dietary Supplement:
• Glytactin
Glycomacropeptide based protein substitute for dietary treatment of PKU
• L-AA
Amino acid based protein substitute for dietary treatment of PKU

Locations

Country	Name	City	State
United Kingdom	Birmingham Women and Children's Hospital	Birmingham	West Midlands
United Kingdom	Great Ormond Street Hospital for Children	London

Sponsors (3)

Lead Sponsor	Collaborator
Ajinomoto Co., Inc.	Birmingham Children's Hospital, Great Ormond Street Hospital for Children NHS Foundation Trust

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in blood Phe in	Change in blood Phe in subjects ingesting purified GMP-AA-protein substitute compared with the change when ingesting L-AA-protein substitute	16 weeks