PhenylketonuriasNCT06332105
Evaluation of Low Phenylalanine Formulas
A study testing Glytactin and L-AA in people with Phenylketonurias, run by Ajinomoto Co., Inc. at 2 sites in United Kingdom.
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). Read more about this study
- Who is it for?
- Ages 3 to 16 years
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- Glytactin and L-AA
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 19 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- 30 Jan 2023to Apr 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- • Milk protein allergy
- Pregnancy
- Severe medical diagnosis not related to PKU
- Treatment with Sapropterin hydrochloride (KUVAN)
Trial locations and contacts
2 sites in 1 country.
United Kingdom2 sites
Birmingham Women and Children's Hospital
Birmingham, West Midlands, B4 6NH
Great Ormond Street Hospital for Children
London, WC1N 3JH
About this study
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
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.
Read the 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
- Being tested
- Glytactin, L-AA
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GlytactinDietary supplement
Also called Glytactin Build 20 Flavours, Glytactin Ready To Drink (RTD) 15 Lite, Glytactin Build 10
Glycomacropeptide based protein substitute for dietary treatment of PKU
L-AADietary supplement
Amino acid based protein substitute for dietary treatment of PKU
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Dietary Supplement: L-AA |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Mar 2024
- Study start30 Jan 2023
- Primary completionJan 2025 (estimated)
- Study completionApr 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriasStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- 2-stage, 15-week randomized crossover trial.
- Masking
- None (open label)
- Enrolment
- 19 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ajinomoto Co., Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Birmingham Children's Hospital, Great Ormond Street Hospital for Children NHS Foundation Trust
- Responsible party
- Sponsor
- Investigators
- Anita MacDonald, BSc PhD, Principal investigator, Birmingham Women and Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06332105
- Sponsor's protocol ID
- CUK001
- Status last verified
- Apr 2024
- First submitted
- 20 Mar 2024
- Last updated
- 15 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06332105
- Acronym / sponsor ID
- CUK001
- Conditions
- Phenylketonurias
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 19 participants (estimated)
- Lead sponsor
- Ajinomoto Co., Inc.Industry (pharma, biotech, devices)
- Collaborators
- Birmingham Children's Hospital, Great Ormond Street Hospital for Children NHS Foundation Trust
- Study start
- 30 Jan 2023 (actual)
- Primary completion
- Jan 2025 (estimated)
- Study completion
- Apr 2025 (estimated)
- First posted
- 27 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 2-stage, 15-week randomized crossover trial.
| Arm | Type | Receives |
|---|---|---|
| Sequence AB: GMP-AA -> L-AA | Active comparator | Dietary Supplement: GlytactinAfter 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. |
| Sequence BA: L-AA -> GMP-AA | Placebo comparator | Dietary Supplement: L-AAAfter 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. |
Who may qualify
- Ages
- 3 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (4 listed)
- • Milk protein allergy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GlytactinAlso: Glytactin Build 20 Flavours, Glytactin Ready To Drink (RTD) 15 Lite, Glytactin Build 10Glycomacropeptide based protein substitute for dietary treatment of PKU | Dietary supplement | Sequence AB: GMP-AA -> L-AA |
| L-AAAmino acid based protein substitute for dietary treatment of PKU | Dietary supplement | Sequence BA: L-AA -> GMP-AA |
Primary outcome
Change in blood Phe in
Time frame: 16 weeks
Change in blood Phe in subjects ingesting purified GMP-AA-protein substitute compared with the change when ingesting L-AA-protein substitute
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anita MacDonald, BSc PhD principal investigator
Birmingham Women and Children's Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Glytactin and L-AA in people with Phenylketonurias, run by Ajinomoto Co., Inc. at 2 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 15 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in blood Phe in (time frame: 16 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Apr 2024.