PKU EASY Microtabs Plus - Acceptability and Tolerance

NCT06694662 | Completed

• 1 other identifier: PKU-004
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Phenylketonuria (PKU)

Keywords

PKU; Microtabs; protein substitute; amino acid

Outcome Measures

Primary Outcomes (3)

• Change in GI tolerance (participant)

  Gastrointestinal tolerance assessed using symptom and severity questionnaire completed by the participants. Severity of symptoms will be rated on a scale of 0 - 3, with 0 being no symptoms and 3 being severe or very troublesome symptoms.

  Week 0, week 1

• Rate of patient compliance with consumption of the prescribed volume of study product

  Assessed via participant diary regarding study product intake. The quantity of study product actually consumed vs the prescribed amount will be recorded. The reason for not consuming the full dose will be recorded by the patient/parent/guardian as free text.

  Week 1

• Change in product acceptability

  Assessed via a product acceptability questionnaire completed by the participant/parent/guardian. Organoleptic properties, such as taste and texture, of the study product will be assessed on a rating scale of terrible to great.

  Week 1

Study Arms (1)

Participant Group

EXPERIMENTAL

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.

Dietary Supplement: PKU EASY Microtabs Plus

Interventions

PKU EASY Microtabs Plus 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.

Participant Group

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Galen Limited: lead
• Birmingham Women's and Children's NHS Foundation Trust: collaborator

Study Sites (1)

Birmingham Children's Hospital

Birmingham, UK, B4 6NH, United Kingdom

Location

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Anita MacDonald, Ph.D.

  Birmingham Women's and Children's NHS Foundation Trust

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 12, 2024
• First Posted: November 19, 2024
• Study Start: March 15, 2025
• Primary Completion: August 13, 2025
• Study Completion: August 13, 2025
• Last Updated: November 26, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)