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Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.

Primary Purpose

Phenylketonurias

Status
Recruiting
Phase
Not Applicable
Locations
United Kingdom
Study Type
Interventional
Intervention
PKU GOLIKE
Sponsored by
APR Applied Pharma Research s.a.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Phenylketonurias

Eligibility Criteria

5 Years - 16 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Male and female PKU patients ≥5 years and ≤16 years of age.
  2. Patients diagnosed with PKU via new born screening.
  3. Taking a Phe free/low Phe protein substitute
  4. On a low phenylalanine diet .
  5. Absence of neurological deficiencies.
  6. Adherence with dietary management and protein substitute.
  7. Able to understand and comply with the requirements of the investigation and sign the Informed Consent Form/Assent form.

Exclusion Criteria:

  1. Age <5 years old and >16 years old.
  2. Patients with mild PKU or HPA.
  3. On sapropterin therapy.
  4. Patients with late diagnosis of PKU and neurological problems.
  5. History of hypersensitivity to any excipients/components of the investigational product.
  6. Pregnancy or breastfeeding during the study.
  7. Any moderate to severe acute illness which in the opinion of the Investigator would interfere with the study procedures or study outcome.
  8. History of poor co-operation, non-adherence with dietary management, or poor adherence to investigation procedures.
  9. Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.

Sites / Locations

  • Birmingham Children's Hospital Steelhouse LaneRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Other

Other

Arm Label

1st PKU GOLIKE 2nd AA protein substitute

1st AA protein substitute 2nd PKU GOLIKE

Arm Description

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.

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

Outcomes

Primary Outcome Measures

Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast
Blood Phe after administration of PKU GOLIKE and after amino acid protein substitute collected before breakfast

Secondary Outcome Measures

Dosage of Tyr in blood (umol/L) with dried blood spots before breakfast
Dosage of blood branch chain amino acids (BCAA)(umol/L) before breakfast
Dosage of Urea (mmol/L) and creatinine (umol/L) in urine
Questionnaire on usual sleep habits
Questionnaire with open questions
Palatability questionnaire on days 7 or 28
Questionnaire with questions on product palatability

Full Information

First Posted
June 17, 2022
Last Updated
October 9, 2023
Sponsor
APR Applied Pharma Research s.a.
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1. Study Identification

Unique Protocol Identification Number
NCT05487378
Brief Title
Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
Official Title
Randomised Investigation to Evaluate Phe Fluctuation in PKU Patients Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 30, 2023 (Actual)
Primary Completion Date
March 2024 (Anticipated)
Study Completion Date
June 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
APR Applied Pharma Research s.a.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
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.
Detailed Description
16 children with PKU who currently take a Phe-free/low Phe protein substitute (3 or 4 doses/day) will be recruited. Subjects will replace the last daily dose of their usual protein substitute with the study product for 7 days of the 28 day trial (either days 1-7 or days 22-28 based on random allocation). On the remaining study days, subjects will take an amino acid based protein substitute for all daily doses. There will be a 2 week washout period between study arms where subjects will take their usual protein substitute. The amount of study product prescribed will be calculated to provide the same amount of protein as their usual protein substitute. The last protein substitute (PS) dose of the day (amino acids or study product) will need to be taken between 7-9pm to allow an 8-10 hour fasting period overnight. Three finger prick blood spots will be collected and analysed for phenylalanine, tyrosine and BCAA at 5am, 6am and 7am on days -1, 0, 6, 7, 20, 21, 27 and 28. For all subjects, a second void urine sample will be collected on days 0, 7, 21 and 28 for analysis of urea and creatinine. A quality of sleep questionnaire will be completed by subjects or their carers on days 0, 7, 21 and 28 and a 24 hour food diary on days -1, 0, 6, 7, 20, 21, 27 and 28. A palatability questionnaire will be completed by subjects or their carers on days 7 or 28 (at the end of the period with PKU GOLIKE, if Bars or Krunches are used). Subject visits will be on days -2 (enrolment), 0, 7, 21 and 28 where the research dietitian will collect urine samples, blood spots, questionnaires and diaries.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Phenylketonurias

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Crossover Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
16 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
1st PKU GOLIKE 2nd AA protein substitute
Arm Type
Other
Arm Description
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.
Arm Title
1st AA protein substitute 2nd PKU GOLIKE
Arm Type
Other
Arm Description
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
Intervention Type
Dietary Supplement
Intervention Name(s)
PKU GOLIKE
Other Intervention Name(s)
PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU.
Intervention Description
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.
Primary Outcome Measure Information:
Title
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
Title
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
Title
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
Title
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
Title
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
Title
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
Title
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
Title
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
Title
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
Title
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
Title
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
Title
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
Secondary Outcome Measure Information:
Title
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
Title
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
Title
Dosage of Urea (mmol/L) and creatinine (umol/L) in urine
Time Frame
Days 0, 7, 21 and 28
Title
Questionnaire on usual sleep habits
Description
Questionnaire with open questions
Time Frame
Day 0, 7,21,28
Title
Palatability questionnaire on days 7 or 28
Description
Questionnaire with questions on product palatability
Time Frame
Day 7 and 28

10. Eligibility

Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Giorgio Reiner
Phone
+41.91.695702
Email
giorgio.reiner@apr.ch
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Anita MacDonald, Pr.
Organizational Affiliation
Birmingham Children's Hospital, Steelhouse Lane, Birmingham B4 6NH
Official's Role
Principal Investigator
Facility Information:
Facility Name
Birmingham Children's Hospital Steelhouse Lane
City
Birmingham
ZIP/Postal Code
B4 6NH
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Anita MacDonald, Dietitian
Phone
01213338024
Email
anita.macdonald@nhs.net

12. IPD Sharing Statement

Learn more about this trial

Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.

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