Phenylketonuria Clinical Trial
— PKU StartOfficial title:
A Study to Evaluate the Acceptability of a New Phenylalanine Free Infant Formula for Use in the Dietary Management of Phenylketonuria in Infants From Birth to 2 Year of Age With Regard to Product Tolerance and Adherence.
Verified date | February 2024 |
Source | Vitaflo International, Ltd |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
To evaluate the acceptability, tolerance and effect on metabolic control of PKU Start, a new Phe free protein substitute for the dietary management of PKU in infants from birth.
Status | Completed |
Enrollment | 10 |
Est. completion date | September 15, 2017 |
Est. primary completion date | September 15, 2017 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 4 Weeks to 2 Years |
Eligibility | Inclusion Criteria: 1. A diagnosis of classical or severe PKU on new-born screening (For the purposes of this study, 'severe' is defined as phe concentrations between 120 and 600 µmol/L at diagnosis) 2. Taking a minimum of one (1) feed of a Phe-free infant formula 3. A minimum period of four (4) weeks from the time of diagnosis to initial approach to parents Exclusion Criteria: 1. Diagnosed with mild PKU or hyperphenylalaninaemia (For the purposes of this study, 'mild' is defined as phe concentrations between 120 and 600 µmol/L at diagnosis) 2. Diagnosis of a congenital condition |
Country | Name | City | State |
---|---|---|---|
United Kingdom | Birmingham Children's Hospital NHS Foundation Trust | Birmingham | West Midlands |
United Kingdom | Bradford Teaching Hospitals NHS Foundation Trust | Bradford | West Yorkshire |
United Kingdom | NHS Greater Glasgow and Clyde | Glasgow | Lanarkshire |
Lead Sponsor | Collaborator |
---|---|
Vitaflo International, Ltd |
United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Product compliance daily diary | Quantitative assessments from subject questionnaires that allow evaluation of compliance with the study product, i.e. actual versus prescribed intake. | Days 1-28 | |
Primary | GI tolerance daily diary | Qualitative assessments from subject questionnaires that allow evaluation of the gastro-intestinal tolerance of the study product. | Days 1-28 | |
Primary | Ease of use questionnaire | Qualitative assessment from subject questionnaire that allows evaluation of the ease of use of the study product. | Day 29 | |
Primary | Daily phenylalanine control | Collection of quantitative data regarding phenylalanine control using routine biochemical testing | Days 1-28 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT05099640 -
A Study of PTC923 in Participants With Phenylketonuria
|
Phase 3 | |
Completed |
NCT01924026 -
Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study
|
N/A | |
Completed |
NCT01428258 -
Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria
|
N/A | |
Completed |
NCT00925054 -
Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU
|
Phase 2 | |
Completed |
NCT00778206 -
PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
|
||
Recruiting |
NCT05948020 -
Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria
|
N/A | |
Recruiting |
NCT05781399 -
First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria
|
Phase 1 | |
Completed |
NCT02555579 -
Simplified Diet Approach in Phenylketonuria
|
N/A | |
Completed |
NCT03097250 -
MRI Spectroscopy and Neuropsychological Functioning in Phenylketonuria
|
||
Completed |
NCT01965691 -
Protein Requirements in Children With Phenylketonuria (PKU)
|
N/A | |
Completed |
NCT01965912 -
Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
|
Phase 4 | |
Completed |
NCT00789568 -
A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
|
Phase 1 | |
Completed |
NCT00688844 -
Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
|
N/A | |
Terminated |
NCT01465100 -
Liver Cell Transplant for Phenylketonuria
|
Phase 1/Phase 2 | |
Completed |
NCT01732471 -
Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria
|
Phase 3 | |
Completed |
NCT04879277 -
Study of Low-grade Systemic Inflammation in Adult Patients With Phenylketonuria
|
N/A | |
Completed |
NCT02176603 -
Observational Study of Endothelial Dysfunction in Phenylketonuria
|
N/A | |
Terminated |
NCT01904708 -
Moderate Intensity Exercise and Phenylketonuria
|
N/A | |
Completed |
NCT01869972 -
Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
|
N/A | |
Completed |
NCT01819727 -
An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
|
Phase 3 |