- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05487378
Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
Randomised Investigation to Evaluate Phe Fluctuation in PKU Patients Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
This is a 2 arm, randomised, controlled, cross-over study in 16 children with PKU. Subjects who are currently taking a Phe free/low Phe protein substitute will be recruited for a 31-day trial.
Patients will be randomised to receive:
- The study product for 7 days as their last dose of protein substitute for the day (at least one sachet with 15g PE) in an amount equivalent to their usual protein substitute PE; or
- An amino acid protein substitute for all daily doses for 7 days; followed by a 2-week washout period on their usual protein substitute,
and then 7 days of the other study arm.
During this time, patients/caregivers will be asked to:
- Collect 3 finger prick blood spots on days -1, 0, 6, 7, 20, 21, 27 and 28.
- Collect urine sample, second void of the day on days 0, 7, 21 and 28.
- Complete a questionnaire on sleep quality on day 0, 7, 21 and 28.
- Complete a 24 hour food diary on days -1, 0, 6, 7, 20, 21, 27 and 28.
APR will supply the study product for participants free of charge.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Estimated)
Phase
- Not Applicable
Contacts and Locations
Study Contact
- Name: Giorgio Reiner
- Phone Number: +41.91.695702
- Email: giorgio.reiner@apr.ch
Study Locations
-
-
-
Birmingham, United Kingdom, B4 6NH
- Recruiting
- Birmingham Children's Hospital Steelhouse Lane
-
Contact:
- Anita MacDonald, Dietitian
- Phone Number: 01213338024
- Email: anita.macdonald@nhs.net
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Male and female PKU patients ≥5 years and ≤16 years of age.
- Patients diagnosed with PKU via new born screening.
- Taking a Phe free/low Phe protein substitute
- On a low phenylalanine diet .
- Absence of neurological deficiencies.
- Adherence with dietary management and protein substitute.
- Able to understand and comply with the requirements of the investigation and sign the Informed Consent Form/Assent form.
Exclusion Criteria:
- Age <5 years old and >16 years old.
- Patients with mild PKU or HPA.
- On sapropterin therapy.
- Patients with late diagnosis of PKU and neurological problems.
- History of hypersensitivity to any excipients/components of the investigational product.
- Pregnancy or breastfeeding during the study.
- Any moderate to severe acute illness which in the opinion of the Investigator would interfere with the study procedures or study outcome.
- History of poor co-operation, non-adherence with dietary management, or poor adherence to investigation procedures.
- Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Other: 1st PKU GOLIKE 2nd AA protein substitute
7 days with usual protein substitute and PKU GOLIKE as their last dose of protein substitute for the day (at least one sachet with15g PE) in an amount equivalent to their usual protein substitute PE followed by a 2-week washout period on their usual protein substitute, and then 7 days with AA protein substitute for all daily doses.
|
AA protein Substitute for the dietary management of PKU, PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU.
Other Names:
|
Other: 1st AA protein substitute 2nd PKU GOLIKE
7 days with AA protein substitute for all daily doses followed by a 2-week washout period on their usual protein substitute, and then 7 days with usual protein substitute and PKU GOLIKE as their last dose of protein substitute for the day (at least one sachet with15g PE) in an amount equivalent to their usual protein substitute PE
|
AA protein Substitute for the dietary management of PKU, PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Time Frame: At 5:00 am on day 6
|
At 5:00 am on day 6
|
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Time Frame: At 5:00 am on day 7
|
At 5:00 am on day 7
|
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Time Frame: At 5:00 am on day 27
|
At 5:00 am on day 27
|
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Time Frame: At 5:00 am on day 28
|
At 5:00 am on day 28
|
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Time Frame: At 6:00 am on day 6
|
At 6:00 am on day 6
|
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Time Frame: At 6:00 am on day 7
|
At 6:00 am on day 7
|
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Time Frame: At 6:00 am on day 27
|
At 6:00 am on day 27
|
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Time Frame: At 6:00 am on day 28
|
At 6:00 am on day 28
|
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Time Frame: At 7:00 am on day 6
|
At 7:00 am on day 6
|
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Time Frame: At 7:00 am on day 7
|
At 7:00 am on day 7
|
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Time Frame: At 7:00 am on day 27
|
At 7:00 am on day 27
|
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Time Frame: At 7:00 am on day 28
|
At 7:00 am on day 28
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Dosage of Tyr in blood (umol/L) with dried blood spots before breakfast
Time Frame: At 5:00, 6:00, 7:00 am on day 6,7,27,28
|
At 5:00, 6:00, 7:00 am on day 6,7,27,28
|
|
Dosage of blood branch chain amino acids (BCAA)(umol/L) before breakfast
Time Frame: At 5:00, 6:00 and 7:00am on day 6,7,27,28
|
At 5:00, 6:00 and 7:00am on day 6,7,27,28
|
|
Dosage of Urea (mmol/L) and creatinine (umol/L) in urine
Time Frame: Days 0, 7, 21 and 28
|
Days 0, 7, 21 and 28
|
|
Questionnaire on usual sleep habits
Time Frame: Day 0, 7,21,28
|
Questionnaire with open questions
|
Day 0, 7,21,28
|
Palatability questionnaire on days 7 or 28
Time Frame: Day 7 and 28
|
Questionnaire with questions on product palatability
|
Day 7 and 28
|
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Anita MacDonald, Pr., Birmingham Children's Hospital, Steelhouse Lane, Birmingham B4 6NH
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- GLK-UK-2021
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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