XPhe Minis - Acceptability and Tolerance Market Research
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Enrollment
- 10
- Locations
- 1
- Primary Endpoint
- Daily compliance
Study Overview
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.
Study Design
- Study Type
- Observational
- Observational Model
- Case Only
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 7 Years to 18 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Diagnosis of PKU or PKU variant requiring a phenylalanine-free protein substitute.
- •Subjects who are already taking a phenylalanine-free protein substitute and are willing to try the study product for 7 days.
- •Children aged 7 years and over.
- •Written informed consent obtained from parental caregiver.
Exclusion Criteria
- •Presence of serious concurrent illness
- •Lead Dietitian's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
- •Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
- •Any children having taken antibiotics over the previous 2 weeks leading up to the study.
- •Children less than 7 years of age.
Outcomes
Primary Outcomes
Daily compliance
Time Frame: 7 days
Compliance, with currently prescribed protein substitute will be assessed at the beginning of the 7-days trial. Usage and compliance with the study product will subsequently assessed daily from days 1-7 using standardised questionnaires, where patients document the amount of consumed study product vs the prescribed doses. Compliance
Secondary Outcomes
- Treatment-Emergent tolerability(7 days)
- Patient Acceptability(7 days)
- Incidence of study product emergent events.(Through study completion an average of 1year)
- Metabolic control(2 days)