Ignite Creation Date:
2025-12-24 @ 3:28 PM
Ignite Modification Date:
2025-12-25 @ 1:44 PM
Study NCT ID:
NCT04375592
Status:
COMPLETED
Last Update Posted:
2024-03-04
First Post:
2020-04-21
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of Phenylketonuria
Sponsor:
metaX Institut fuer Diatetik GmbH
Organization:
Study Overview
Official Title:
XPhe Minis - Acceptability and Tolerance Market Research
Status:
COMPLETED
Status Verified Date:
2024-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this prospective, observational study is to evaluate the tolerability and acceptability of phenylalanine-free protein substitute tablets for young children with PKU aged of 7 years or older.
Detailed Description:
The principle treatment for children with phenylketonuria (PKU) is a low protein diet. Part of this treatment requires the administration of a phenylalanine-free protein substitute in order to meet basic protein requirements for normal growth and development. Several brands of phenylalanine-free protein substitutes are already available in various forms - liquid, semi solid, tablet. However, compliance with taking protein substitutes continues to be a challenge. As a low protein diet is recommended for life, long term compliance is a growing concern. As a result, improving the choice in terms of product type may aid compliance. XPhe minis are phenylalanine-free protein substitute tablets enriched with vitamins, minerals and trace elements designed for children and adults with PKU. It is anticipated that they will be a suitable alternative choice for PKU patients, broadening the variety of protein substitutes they can choose from in order to suit their lifestyle and preferences.
This is a prospective, observational tolerance study in 10 children with PKU. Subjects who are currently taking a second stage protein substitute will be recruited for a 7-day trial, taking the ready-to-use protein substitute tablets to evaluate the tolerability and acceptability of the study product. Therefore, subjects will replace some or all of their usual protein substitute with the new product.
During the 7-days trial subjects or caregivers will be asked to complete a daily questionnaire recording information on: • Usage and compliance • Ease of use and any issues with administration • Any gastro-intestinal side-effects.
A questionnaire will also be completed at the beginning and end of the study that will consider perceptions about taste, appearance, smell, ease of administration; how it is taken; and any other problems or symptoms.
The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute.
Subjects will continue to have weekly finger-prick blood tests as is routine in PKU. The results whilst on the study product will be compared with results whilst on their usual protein substitute.
10 children with PKU will be recruited. When an appropriate subject has been identified, a study information sheet will be sent to the subject or parents/caregivers. They will be invited to request further information about the study if they wish by contacting the Lead Dietitian. Recruitment of each patient will be by written informed consent, which will be completed by the parents/primary caregivers and taken by the Lead Dietitian. Children will also complete an assent/consent form and will receive an information sheet, if considered appropriate for their level of understanding.
This is a prospective, observational tolerance study in 10 children with PKU. Subjects who are currently taking a second stage protein substitute will be recruited for a 7-day trial, taking the ready-to-use protein substitute tablets to evaluate the tolerability and acceptability of the study product. Therefore, subjects will replace some or all of their usual protein substitute with the new product.
During the 7-days trial subjects or caregivers will be asked to complete a daily questionnaire recording information on: • Usage and compliance • Ease of use and any issues with administration • Any gastro-intestinal side-effects.
A questionnaire will also be completed at the beginning and end of the study that will consider perceptions about taste, appearance, smell, ease of administration; how it is taken; and any other problems or symptoms.
The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute.
Subjects will continue to have weekly finger-prick blood tests as is routine in PKU. The results whilst on the study product will be compared with results whilst on their usual protein substitute.
10 children with PKU will be recruited. When an appropriate subject has been identified, a study information sheet will be sent to the subject or parents/caregivers. They will be invited to request further information about the study if they wish by contacting the Lead Dietitian. Recruitment of each patient will be by written informed consent, which will be completed by the parents/primary caregivers and taken by the Lead Dietitian. Children will also complete an assent/consent form and will receive an information sheet, if considered appropriate for their level of understanding.
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: