PKU EASY Microtabs Plus - Acceptability and Tolerance

NCT ID: NCT06694662

Last Updated: 2025-11-26

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-03-15
• Study Completion Date: 2025-08-13

Brief Summary

PEMP-AT is a prospective, single-arm, open-label, 1-week acceptability study to evaluate PKU EASY Microtabs Plus for the dietary management of participants with phenylketonuria (PKU). Up to 10 participants aged 3 years and above will be recruited in a single-centre in the UK.

Detailed Description

PKU is a rare inborn error of metabolism with a prevalence of 1 in 10,000 in the UK population. The mainstay of treatment for most individuals is dietary therapy with a strict protein-restricted diet. We propose to recruit children with a diagnosis of PKU aged 3 years and above.

The study product, PKU EASY Microtabs Plus is a food for special medical purposes (FSMP), as defined by the Delegated Act EU 2016/128, used for the dietary management of Phenylketonuria (PKU).

The study will be investigating the acceptability of PKU EASY Microtabs Plus, as defined by the Advisory Committee on Borderline Substances (ACBS). This includes the following:

participant adherence to recommended intakes gastrointestinal symptoms evaluations of palatability. Each participant will be on the study for 1 week.

The study consists of a 1 week period where participants introduce the study product into their diet. Gastrointestinal tolerance, compliance and product acceptability will be evaluated.

Data collection will be performed using paper data collection forms completed by the investigators at the baseline and end of study visits. Patients/parents/guardians, as appropriate, will also complete a brief daily questionnaire on each of the 7 study days. There will also be a questionnaire completed by the patient/parents/guardians over the course of the study, in relation to protein substitute intake, gastrointestinal tolerance, and product acceptability.

Conditions

Phenylketonuria (PKU)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Participant Group

All participants will be assessed by their dietitian and prescribed an appropriate amount of the study product, PKU EASY Microtabs Plus, to manage their phenylketonuria.

Group Type: EXPERIMENTAL

PKU EASY Microtabs Plus

Intervention Type: DIETARY_SUPPLEMENT

PKU EASY Microtabs Plus is a food for special medical purposes (FSMP). This product is for use in the dietary management of Phenylketonuria (PKU) in patients from 3 years of age upwards.

PKU EASY Microtabs Plus is a microtablet form, slow-release, phenylalanine-free protein substitute containing a blend of essential and non-essential amino acids, vitamins, minerals and trace elements.

Interventions

PKU EASY Microtabs Plus

PKU EASY Microtabs Plus is a food for special medical purposes (FSMP). This product is for use in the dietary management of Phenylketonuria (PKU) in patients from 3 years of age upwards.

PKU EASY Microtabs Plus is a microtablet form, slow-release, phenylalanine-free protein substitute containing a blend of essential and non-essential amino acids, vitamins, minerals and trace elements.

Intervention Type: DIETARY_SUPPLEMENT

Eligibility Criteria

Inclusion Criteria

• Diagnosis of PKU requiring an appropriate protein substitute
• Subjects who are already taking an appropriate protein substitute and are willing to try the study product for 7 days
• Subjects aged 3 years and above
• Written informed consent obtained from subject or parent/legal guardian, depending on subject age

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, with the exception of sapropterin
• Any subject having taken antibiotics over the previous 2 weeks leading up to the study
• Subjects less than 3 years of age

• Minimum Eligible Age: 3 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Birmingham Women's and Children's NHS Foundation Trust

OTHER

Sponsor Role: collaborator

Galen Limited

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Anita MacDonald, Ph.D.

Role: PRINCIPAL_INVESTIGATOR

Birmingham Women's and Children's NHS Foundation Trust

Locations

Birmingham Children's Hospital

Birmingham, UK, United Kingdom

Countries

United Kingdom

Other Identifiers

PKU-004

Identifier Type: -

Identifier Source: org_study_id