Evaluation of Low Phenylalanine Formulas

NCT06332105 | Active Not Recruiting

• 1 other identifier: CUK001
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Phenylketonurias

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (2)

Sequence AB: GMP-AA -> L-AA

ACTIVE COMPARATOR

After an initial washout period of 2 weeks, participants randomised to Sequence AB will consume GMP-AA for 3 months whilst keeping a food diary and sending blood spots for twice weekly analysis. At the end of 3 months there is a further 2 week washout period with L-AA, after which the participant will consume L-AA for 3 months whilst keeping a food diary and sending blood spots for twice weekly analysis.

Dietary Supplement: Glytactin

Sequence BA: L-AA -> GMP-AA

PLACEBO COMPARATOR

After an initial washout period of 2 weeks, participants randomised to Sequence BA will consume L-AA for 3 months whilst keeping a food diary and sending blood spots for twice weekly analysis. At the end of 3 months there is a further 2 week washout period with L-AA, after which the participant will consume GMP-AA for 3 months whilst keeping a food diary and sending blood spots for twice weekly analysis.

Dietary Supplement: L-AA

Interventions

Glytactin DIETARY_SUPPLEMENT

Glycomacropeptide based protein substitute for dietary treatment of PKU

Also known as: Glytactin Build 20 Flavours, Glytactin Ready To Drink (RTD) 15 Lite, Glytactin Build 10

Sequence AB: GMP-AA -> L-AA

L-AA DIETARY_SUPPLEMENT

Amino acid based protein substitute for dietary treatment of PKU

Sequence BA: L-AA -> GMP-AA

Eligibility Criteria

• Age: 3 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• 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

You may not qualify if:

• Milk protein allergy
• Pregnancy
• Severe medical diagnosis not related to PKU
• Treatment with Sapropterin hydrochloride (KUVAN)

Sponsors & Collaborators

• Ajinomoto Co., Inc.: lead
• Birmingham Children's Hospital: collaborator
• Great Ormond Street Hospital for Children NHS Foundation Trust: collaborator

Study Sites (2)

Birmingham Women and Children's Hospital

Birmingham, West Midlands, B4 6NH, United Kingdom

Location

Great Ormond Street Hospital for Children

London, WC1N 3JH, United Kingdom

Location

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Anita MacDonald, BSc PhD

  Birmingham Women and Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 2-stage, 15-week randomized crossover trial.

Study Record Dates

• First Submitted: March 20, 2024
• First Posted: March 27, 2024
• Study Start: January 30, 2023
• Primary Completion: January 1, 2025
• Study Completion: April 1, 2025
• Last Updated: April 15, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

GB (2)