PhenylketonuriasNCT03419819
Evaluation of PKU Sphere
A study testing PKU Sphere in people with Phenylketonurias, run by Vitaflo International, Ltd at 1 site in United States.
Trial summary
A prospective, open-label study of PKU Sphere in patients with PKU following a phenylalanine restricted therapeutic diet. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- PKU Sphere
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 1 siteUnited States
- Study dates
- 1 May 2018to 21 May 2019
- 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of PKU, identified by newborn screening (NBS) and treated since birth.
- Ages 3 years and above (this includes pregnant women who will be assessed on an individual basis by the Co-Principal Investigators).
- Currently or previously consuming a low phenylalanine diet supplemented with a medical food designed for the dietary management of PKU.
- English as primary language.
- Willingly given, written, informed consent from the participant, 18 or more years, or from the parent/caregiver for participants < 18 years.
- Willingly given, written assent (if appropriate) for those < 18 years.
Who cannot join
- Currently consuming 100% of the medical food component of the diet as PKU sphere.
- An inability, in the opinion of the investigator, to comply with the requirements of the protocol.
- Any other type of inherited metabolic disease.
Trial locations and contacts
1 site in 1 country.
United States1 site
Oregon Health and Science University
Portland, Oregon, 97239
About this study
Official title: Evaluation of the Acceptability, Tolerance, and Satiety of PKU Sphere, a Glycomacropeptide (GMP) Based Medical Food in Patients With Phenylketonuria (PKU).
A prospective, open-label study of PKU Sphere in patients with PKU following a phenylalanine restricted therapeutic diet.
Read the detailed description
The rationale for this study is to assess acceptability, adherence and metabolic control in individuals with PKU consuming PKU Sphere, a GMP-based medical food.
The sponsor developed PKU Sphere in response to growing interest in GMP medical foods for the purpose of improving adherence and quality of life by offering an alternative to amino acid based medical foods. PKU Sphere is a powdered, low phenylalanine medical food containing a balanced mix of casein glycomacropeptide (GMP) isolate, essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals and the long chain polyunsaturated fatty acid (LCP); docosahexaenoic acid (DHA). PKU Sphere has been designed for use in the dietary management of Phenylketonuria. It is available in two flavors, Red Berry and Vanilla, packaged in individual serving sachets of 35g and containing 20g protein equivalent (PE).
- Conditions
- Phenylketonurias
- Being tested
- PKU Sphere
- Keywords
- Phenylketonuria, GMP, Sphere, Child, Adult, Phenylalanine, PKU, Glycomacropeptide
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PKU SphereDietary supplement
PKU Sphere is a glycomacropeptide based alternative to amino-acid based products for the dietary management of phenylketonuria.
| Study group | Role | What they receive |
|---|---|---|
| PKU Sphere | Experimental | Phase 1: 1 week To evaluate the acceptability of PKU Sphere during a short-term (1 week) period. Individuals with PKU will aim to consume a minimum of 30% of the medical food component of the diet as PKU Sphere. The amount will be assessed and advised on an individual basis.
Phase 2: 4 weeks To evaluate longer-term acceptability and metabolic control in individuals with PKU consuming an agreed target of 50 - 100% of their medical food component of the diet as PKU Sphere for 4 weeks. Some individuals, particularly young children between the ages of 3 - 6 years, may require a 1 - 3 week build up period to reach target volume which will be assessed on an individual basis. Receives: Dietary Supplement: PKU Sphere |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1: taste
Questionnaire data captured to evaluate taste
Measured: Days 1 - 7.
Phase 1: smell
Questionnaire data captured to evaluate smell
Measured: Days 1 - 7.
Phase 1: texture
Questionnaire data captured to evaluate texture
Measured: Days 1 - 7.
Phase 1: gastrointestinal tolerance
Questionnaires will be completed daily during the 1-week taste test.
Measured: Days 1 - 7.
Phase 1: phenylalanine concentration
Phenylalanine concentrations measured via blood spot analysis.
Measured: Days 1 and 7.
Phase 1: tyrosine concentration
Tyrosine concentrations measured via blood spot analysis.
Measured: Days 1 and 7.
Phase 2: quantitative change in plasma amino acids at day 28
Compare comprehensive plasma amino acid profiles at baseline and end of the trial.
Measured: Phase 2, day 1 and day 28.
Phase 2: gastrointestinal tolerance
Questionnaires will be completed daily throughout Phase 2.
Measured: Phase 2, days 1 - 28.
Phase 2: change in 3-day diet record at end of study
A 3-day diet record will be completed in the first 3 days and the last 3 days for comparison.
Measured: Phase 2, days 1 - 3 and days 26 - 28.
Phase 2: change in anthropometry at end of study
Participants will be weighed and measured, a growth chart plotted (children only), and BMI calculated
Measured: Phase 2, days 1 and 28.
Phase 2: phenylalanine concentration
Phenylalanine concentrations measured via blood spot analysis.
Measured: Twice weekly for four weeks during phase 2
Phase 2: tyrosine concentration
Tyrosine concentrations measured via blood spot analysis.
Measured: Twice weekly for four weeks during phase 2
Phase 2: questionnaire data captured to evaluate change in taste perception
Data captured to evaluate taste
Measured: Phase 2, days 14 and 28.
Phase 2: questionnaire data captured to evaluate change in smell perception
Data captured to evaluate smell
Measured: Phase 2, days 14 and 28.
Phase 2: questionnaire data captured to evaluate change in texture perception
Data captured to evaluate texture
Measured: Phase 2, days 14 and 28.
Secondary outcomes (2)
Phase 1: questoinnaire evaluating subjective measures of satiety
To evaluate if PKU Sphere increases subjective measures of satiety
Measured: Phase 1, days 1 - 7.
Phase 2: questoinnaire evaluating subjective measures of satiety
To evaluate if PKU Sphere increases subjective measures of satiety
Measured: Phase 2, days 1, 14 and 28.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2018
- Study start1 May 2018
- Primary completion21 May 2019
- Study completion21 May 2019
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
- Not applicable
- Intervention model
- Single group
- Model description
- Two phase study consisting of a 1 week taste introduction period followed by a 4 week period assessing acceptability and metabolic control.
- Masking
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vitaflo International, Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Oregon Health and Science University
- Responsible party
- Sponsor
- Investigators
- Sandra Van Calcar, PhD, Principal investigator, Oregon Health and Science UniversityJoyanna Hansen, PhD, Principal investigator, Oregon Health and Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03419819
- Sponsor's protocol ID
- SCT-PKUS-2016-11-28
- Status last verified
- Feb 2024
- First submitted
- 3 Jan 2018
- Last updated
- 16 Feb 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 16 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Acceptability, Tolerance, and Satiety of PKU Sphere, a Glycomacropeptide (GMP) Based Medical Food in Patients With Phenylketonuria (PKU).
- ClinicalTrials.gov ID
- NCT03419819
- Acronym / sponsor ID
- SCT-PKUS-2016-11-28
- Conditions
- Phenylketonurias
- Keywords
- Phenylketonuria, GMP, Sphere, Child, Adult, Phenylalanine, PKU, Glycomacropeptide
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Vitaflo International, LtdIndustry (pharma, biotech, devices)
- Collaborators
- Oregon Health and Science University
- Study start
- 1 May 2018 (actual)
- Primary completion
- 21 May 2019 (actual)
- Study completion
- 21 May 2019 (actual)
- First posted
- 5 Feb 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.
A prospective, open-label study of PKU Sphere in patients with PKU following a phenylalanine restricted therapeutic diet.
Detailed description
The rationale for this study is to assess acceptability, adherence and metabolic control in individuals with PKU consuming PKU Sphere, a GMP-based medical food.
The sponsor developed PKU Sphere in response to growing interest in GMP medical foods for the purpose of improving adherence and quality of life by offering an alternative to amino acid based medical foods. PKU Sphere is a powdered, low phenylalanine medical food containing a balanced mix of casein glycomacropeptide (GMP) isolate, essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals and the long chain polyunsaturated fatty acid (LCP); docosahexaenoic acid (DHA). PKU Sphere has been designed for use in the dietary management of Phenylketonuria. It is available in two flavors, Red Berry and Vanilla, packaged in individual serving sachets of 35g and containing 20g protein equivalent (PE).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Two phase study consisting of a 1 week taste introduction period followed by a 4 week period assessing acceptability and metabolic control.
| Arm | Type | Receives |
|---|---|---|
| PKU Sphere | Experimental | Dietary Supplement: PKU SpherePhase 1: 1 week To evaluate the acceptability of PKU Sphere during a short-term (1 week) period. Individuals with PKU will aim to consume a minimum of 30% of the medical food component of the diet as PKU Sphere. The amount will be assessed and advised on an individual basis. Phase 2: 4 weeks To evaluate longer-term acceptability and metabolic control in individuals with PKU consuming an agreed target of 50 - 100% of their medical food component of the diet as PKU Sphere for 4 weeks. Some individuals, particularly young children between the ages of 3 - 6 years, may require a 1 - 3 week build up period to reach target volume which will be assessed on an individual basis. |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of PKU, identified by newborn screening (NBS) and treated since birth.
- Ages 3 years and above (this includes pregnant women who will be assessed on an individual basis by the Co-Principal Investigators).
- Currently or previously consuming a low phenylalanine diet supplemented with a medical food designed for the dietary management of PKU.
- English as primary language.
- Willingly given, written, informed consent from the participant, 18 or more years, or from the parent/caregiver for participants < 18 years.
Key exclusion criteria (3 listed)
- Currently consuming 100% of the medical food component of the diet as PKU sphere.
- An inability, in the opinion of the investigator, to comply with the requirements of the protocol.
- Any other type of inherited metabolic disease.
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 SpherePKU Sphere is a glycomacropeptide based alternative to amino-acid based products for the dietary management of phenylketonuria. | Dietary supplement | PKU Sphere |
Primary outcome
Phase 1: taste
Time frame: Days 1 - 7.
Questionnaire data captured to evaluate taste
Phase 1: smell
Time frame: Days 1 - 7.
Questionnaire data captured to evaluate smell
Phase 1: texture
Time frame: Days 1 - 7.
Questionnaire data captured to evaluate texture
Phase 1: gastrointestinal tolerance
Time frame: Days 1 - 7.
Questionnaires will be completed daily during the 1-week taste test.
Phase 1: phenylalanine concentration
Time frame: Days 1 and 7.
Phenylalanine concentrations measured via blood spot analysis.
Phase 1: tyrosine concentration
Time frame: Days 1 and 7.
Tyrosine concentrations measured via blood spot analysis.
Phase 2: quantitative change in plasma amino acids at day 28
Time frame: Phase 2, day 1 and day 28.
Compare comprehensive plasma amino acid profiles at baseline and end of the trial.
Phase 2: gastrointestinal tolerance
Time frame: Phase 2, days 1 - 28.
Questionnaires will be completed daily throughout Phase 2.
Phase 2: change in 3-day diet record at end of study
Time frame: Phase 2, days 1 - 3 and days 26 - 28.
A 3-day diet record will be completed in the first 3 days and the last 3 days for comparison.
Phase 2: change in anthropometry at end of study
Time frame: Phase 2, days 1 and 28.
Participants will be weighed and measured, a growth chart plotted (children only), and BMI calculated
Phase 2: phenylalanine concentration
Time frame: Twice weekly for four weeks during phase 2
Phenylalanine concentrations measured via blood spot analysis.
Phase 2: tyrosine concentration
Time frame: Twice weekly for four weeks during phase 2
Tyrosine concentrations measured via blood spot analysis.
Phase 2: questionnaire data captured to evaluate change in taste perception
Time frame: Phase 2, days 14 and 28.
Data captured to evaluate taste
Phase 2: questionnaire data captured to evaluate change in smell perception
Time frame: Phase 2, days 14 and 28.
Data captured to evaluate smell
Phase 2: questionnaire data captured to evaluate change in texture perception
Time frame: Phase 2, days 14 and 28.
Data captured to evaluate texture
2 secondary outcomes
Phase 1: questoinnaire evaluating subjective measures of satiety
Time frame: Phase 1, days 1 - 7.
Phase 2: questoinnaire evaluating subjective measures of satiety
Time frame: Phase 2, days 1, 14 and 28.
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 16 Feb 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.
Sandra Van Calcar, PhD principal investigator
Oregon Health and Science University
Joyanna Hansen, PhD principal investigator
Oregon Health and Science University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PKU Sphere in people with Phenylketonurias, run by Vitaflo International, Ltd at 1 site in United States.
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 6 inclusion and 3 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 16 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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?
Phase 1: taste (time frame: Days 1 - 7.). The study lists 15 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 16 Feb 2024.