PhenylketonuriasESSENTIALNCT03777826
Effects of Synergy on Nutrient Intake and Acceptability in Phenylketonuria (PKU)
A study testing PKU Synergy in people with Phenylketonurias, run by Nutricia Research at 3 sites in 2 countries.
Trial summary
Phenylketonuria (PKU) is a rare inherited metabolic disorder, where subjects are born with a genetic deficiency in the phenylalanine hydroxylase enzyme (PAH), which leaves them unable to convert Phenylalanine (Phe) into Tyrosine (Tyr). Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- PKU Synergy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 26 Jun 2019to 27 May 2021
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- PKU subjects identified by newborn screening and started low-Phe diet before 3 months of age
- PKU subjects (with or without current AAM use) with an increased Phe-tolerance/intake due to:
- Mild PKU phenotype
- BH4 treatment
- If treated with BH4, PKU subjects should be on a stable BH4 treatment for at least 26 consecutive weeks up to start test product intake
- Age≥12 years
Show all 12Show fewer
- If subjects (irrespective whether BH4 users or mild PKU) use amino-acid mixture(s; AAM), then a maximum of 25 Protein Equivalents (PE) derived from the AAM per day applies and usage on a daily basis for at least 26 consecutive weeks up to Visit 1
- If subjects (irrespective whether BH4 users or mild PKU) use AAMs they should be capable and willing to substitute their current AAM(s; maximum of 25 PE per day) with one portion of the test product per day
- If subjects (irrespective whether BH4 users or mild PKU) use omega-3, antioxidant, and/or vitamin supplements, to stop usage of the supplements and be able and willing to substitute with the test product
- Willing and able to comply with study procedures
- Willing and able to provide informed consent (and assent in case of minors if required by local law/regulations)
- For women of childbearing potential: not to have the intention to become pregnant during the study
Who cannot join
- For women: Currently pregnant or lactating
- Current or prior use of the test product within six weeks prior to entry into the study
- Concurrent conditions (including renal failure and severe hepatic failure) and medication that could interfere with participation, outcome parameters or safety (as determined by Investigator)
- Psychotropic medication (i.e. medication affecting the nervous system) and inotropic medication
- Any condition creating high risk of poor compliance with study
- Participation in any other studies involving investigational or marketed products concomitantly or within six weeks prior to entry into the study. Except for studies related to Kuvan® (synthetic tetrahydrobiopterin (BH4)) without diagnostic, therapeutic or experimental intervention.
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
Germany2 sites
University hospital Leipzig
Leipzig
University hospital Münster
Münster
About this study
Official title: An Open-label, Explorative, Post Launch, Multicenter, Multi-country Intervention Study of PKU Synergy (an Amino-acid Mixture) to Evaluate Change in Nutrient Intake in PKU Subjects With an Increased Phe-tolerance/Intake
Phenylketonuria (PKU) is a rare inherited metabolic disorder, where subjects are born with a genetic deficiency in the phenylalanine hydroxylase enzyme (PAH), which leaves them unable to convert Phenylalanine (Phe) into Tyrosine (Tyr). Patients with mild PKU or partly responsive to the drug synthetic tetrahydrobiopterin (BH4) (Kuvan®) can change to a more relaxed diet. However due to difficulty to adapt their diet, these patients are at risk of an imbalanced nutritional status and an insufficient intake of specific micronutrients, essential amino acids and DHA (Docosahexaenoic acid). The study product is designed to improve the nutritional status of the patients.
The study investigates if the nutritional status is indeed improved following 24 week use of the study product, and also the study aims to evaluate product acceptability.
- Conditions
- Phenylketonurias
- Being tested
- PKU Synergy
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PKU SynergyOther
PKU Synergy is a citrus flavored, powdered amino-acid mixture (containing traces of Phe, 4,3mg per portion; and 20 gr. Protein Equivalent (PE)) with a tailored amino acid and micronutrient profile adapted for the special requirements of HPA/PKU (Hyperphenylalaninemia/Phenylketonuria) subjects over 10 years of age with an increased Phenylalanine-(Phe) tolerance/intake.
| Study group | Role | What they receive |
|---|---|---|
| Open label (1 arm) | Other | Open label use of study product (post-marketing): PKU Synergy Receives: Other: PKU Synergy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Nutrient intake
Change in nutrient intake after 24 weeks by analysis of 3 day diet diary. Nutrients (energy, micronutrients, essential amino acids and DHA) in [(m)/(micro)gram/day] or [mg/day or mg/kg/day]
Measured: 24 weeks
Product acceptability
Product acceptability questionnaire [category/score] [0-10] and [0-5]
Measured: 24 weeks
Secondary outcomes (6)
Compliance
Compliance (product consumption) using daily product intake diary [number of products used per week; per sachet of 33g]
Measured: 24 weeks
Phenylalanine (Phe) levels
Dried bloodspot Phenylalanine (Phe) levels [µmol/L]
Measured: 24 weeks
Blood chemistry: nutritional status
Blood chemistry: levels measured in blood: Full Fatty acid profile [For each FA Erythrocyte Membranes level (%FA)], full Amino acid profile [µmol/L], Vitamin B12 [pM], Vitamin D [ng/ml /nmol/L]; Folic acid [nM]; Selenium [µM]; Iodine [µg/L/ ng/mL]; Calcium [mg/dL]; Zinc [µmol/L]; Iron [µg/L] analyzed in blood samples by central laboratories
Measured: 24 weeks
Anthropometrics: weight
Anthropometrics: Weight [kg]
Measured: 24 weeks
Anthropometrics: height
Anthropometrics: Height [cm or m]
Measured: 24 weeks
Anthropometrics: BMI
Anthropometrics: BMI [kg/m2], calculated from weight/height
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2018
- Study start26 Jun 2019
- Primary completion27 May 2021
- Study completion27 May 2021
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.
- More trials run by Nutricia ResearchThe sponsor's other registered studies.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nutricia Research
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03777826
- Sponsor's protocol ID
- MPR16TA09791
- Acronym
- ESSENTIAL
- Status last verified
- Dec 2021
- First submitted
- 19 Nov 2018
- Last updated
- 9 Dec 2021
- 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 9 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Explorative, Post Launch, Multicenter, Multi-country Intervention Study of PKU Synergy (an Amino-acid Mixture) to Evaluate Change in Nutrient Intake in PKU Subjects With an Increased Phe-tolerance/Intake
- ClinicalTrials.gov ID
- NCT03777826
- Acronym / sponsor ID
- ESSENTIAL / MPR16TA09791
- Conditions
- Phenylketonurias
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Nutricia ResearchIndustry (pharma, biotech, devices)
- Study start
- 26 Jun 2019 (actual)
- Primary completion
- 27 May 2021 (actual)
- Study completion
- 27 May 2021 (actual)
- First posted
- 17 Dec 2018 (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.
Phenylketonuria (PKU) is a rare inherited metabolic disorder, where subjects are born with a genetic deficiency in the phenylalanine hydroxylase enzyme (PAH), which leaves them unable to convert Phenylalanine (Phe) into Tyrosine (Tyr). Patients with mild PKU or partly responsive to the drug synthetic tetrahydrobiopterin (BH4) (Kuvan®) can change to a more relaxed diet. However due to difficulty to adapt their diet, these patients are at risk of an imbalanced nutritional status and an insufficient intake of specific micronutrients, essential amino acids and DHA (Docosahexaenoic acid). The study product is designed to improve the nutritional status of the patients.
The study investigates if the nutritional status is indeed improved following 24 week use of the study product, and also the study aims to evaluate product acceptability.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open label (1 arm) | Other | Other: PKU SynergyOpen label use of study product (post-marketing): PKU Synergy |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- PKU subjects identified by newborn screening and started low-Phe diet before 3 months of age
- PKU subjects (with or without current AAM use) with an increased Phe-tolerance/intake due to:
- If treated with BH4, PKU subjects should be on a stable BH4 treatment for at least 26 consecutive weeks up to start test product intake
- Age≥12 years
- If subjects (irrespective whether BH4 users or mild PKU) use amino-acid mixture(s; AAM), then a maximum of 25 Protein Equivalents (PE) derived from the AAM per day applies and usage on a daily basis for at least 26 consecutive weeks up to Visit 1
Key exclusion criteria (6 listed)
- For women: Currently pregnant or lactating
- Current or prior use of the test product within six weeks prior to entry into the study
- Concurrent conditions (including renal failure and severe hepatic failure) and medication that could interfere with participation, outcome parameters or safety (as determined by Investigator)
- Psychotropic medication (i.e. medication affecting the nervous system) and inotropic medication
- Any condition creating high risk of poor compliance with study
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 |
|---|---|---|
| PKU SynergyPKU Synergy is a citrus flavored, powdered amino-acid mixture (containing traces of Phe, 4,3mg per portion; and 20 gr. Protein Equivalent (PE)) with a tailored amino acid and micronutrient profile adapted for the special requirements of HPA/PKU (Hyperphenylalaninemia/Phenylketonuria) subjects over 10 years of age with an increased Phenylalanine-(Phe) tolerance/intake. | Other | Open label (1 arm) |
Primary outcome
Nutrient intake
Time frame: 24 weeks
Change in nutrient intake after 24 weeks by analysis of 3 day diet diary. Nutrients (energy, micronutrients, essential amino acids and DHA) in [(m)/(micro)gram/day] or [mg/day or mg/kg/day]
Product acceptability
Time frame: 24 weeks
Product acceptability questionnaire [category/score] [0-10] and [0-5]
6 secondary outcomes
Compliance
Time frame: 24 weeks
Phenylalanine (Phe) levels
Time frame: 24 weeks
Blood chemistry: nutritional status
Time frame: 24 weeks
Anthropometrics: weight
Time frame: 24 weeks
Anthropometrics: height
Time frame: 24 weeks
Anthropometrics: BMI
Time frame: 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Dec 2021.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PKU Synergy in people with Phenylketonurias, run by Nutricia Research at 3 sites in 2 countries.
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 12 inclusion and 6 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: finished, as of the sponsor's last update on 9 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Germany, Netherlands. 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. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Nutrient intake (time frame: 24 weeks). The study lists 2 primary outcomes in all. 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 9 Dec 2021.