Evaluation of Low Phenylalanine Formulas

NCT ID: NCT06332105

Last Updated: 2024-04-15

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: NA
• Total Enrollment: 19 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-01-30
• Study Completion Date: 2025-04-30

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.

Detailed 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.

Conditions

Phenylketonurias

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Sequence AB: GMP-AA -> L-AA

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.

Group Type: ACTIVE_COMPARATOR

Glytactin

Intervention Type: DIETARY_SUPPLEMENT

Glycomacropeptide based protein substitute for dietary treatment of PKU

Sequence BA: L-AA -> GMP-AA

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.

Group Type: PLACEBO_COMPARATOR

L-AA

Intervention Type: DIETARY_SUPPLEMENT

Amino acid based protein substitute for dietary treatment of PKU

Interventions

Glytactin

Glycomacropeptide based protein substitute for dietary treatment of PKU

Intervention Type: DIETARY_SUPPLEMENT

L-AA

Amino acid based protein substitute for dietary treatment of PKU

Intervention Type: DIETARY_SUPPLEMENT

Other Intervention Names

Glytactin Build 20 Flavours; Glytactin Ready To Drink (RTD) 15 Lite; Glytactin Build 10

Eligibility Criteria

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)

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Birmingham Children's Hospital

OTHER

Sponsor Role: collaborator

Great Ormond Street Hospital for Children NHS Foundation Trust

OTHER

Sponsor Role: collaborator

Ajinomoto Co., Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Anita MacDonald, BSc PhD

Role: PRINCIPAL_INVESTIGATOR

Birmingham Women and Children's Hospital

Locations

Birmingham Women and Children's Hospital

Birmingham, West Midlands, United Kingdom

Great Ormond Street Hospital for Children

London, , United Kingdom

Countries

United Kingdom

Other Identifiers

CUK001

Identifier Type: -

Identifier Source: org_study_id