Acceptability and Tolerance Market Research of a Slow Release, Phenylalanine-free Protein Substitute for the Dietary Management of Phenylketonuria

NCT07802366 | Not Yet Recruiting

• 1 other identifier: XPhePiccosCT01
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

XPhePiccosMR is a prospective, single-arm, open label, observational study that aims to evaluate the acceptability and tolerability of XPhe Piccos, a slow-release, phenylalanine-free protein substitute in the form of tiny tablets, for use in the dietary management of phenylketonuria (PKU) in children aged three years and older.

Conditions

Phenylketonuria (PKU) and Hyperphenylalaninemia; Brain Diseases, Metabolic, Inborn; Brain Disorder, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Disease, Inborn; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Keywords

PKU; HPA; protein substitute; 7-day trial; slow release

Outcome Measures

Primary Outcomes (1)

• Compliance

  Compliance with currently prescribed protein substitue will be assessed at baseline. 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.

  daily; from day 1 to day 7

Secondary Outcomes (4)

• Treatment-Emergent tolerability

  daily; from day 1 to day 7

• Patient Acceptability

  daily; from day 1 to day 7

• Metabolic Control

  baseline (day 1) and day 7

• Incidence of study product emergent events

  throughout the study; from day 1 to day 7

Study Arms (1)

Slow release protein substitute

EXPERIMENTAL

Dietary Supplement: Slow release, phenylalanine-free protein substitute tablets

Interventions

Slow release, phenylalanine-free protein substitute tablets DIETARY_SUPPLEMENT

Subjects who currently take a concentrated second stage phenylalanine-free protein substitute will be recruited. Subjects will take the study product for 7 days. Subjects will replace some or all of their usual protein substitute with the new product. The amount of study product prescribed will be calculated to provide the same amount of protein as their usual protein substitute.

Slow release protein substitute

Eligibility Criteria

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

You may qualify if:

• Diagnosis of PKU or HPA 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.
• Subjects aged 3 years and above.
• Written informed assent/consent obtained from subject and/or parental caregiver, 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.
• Any subject having taken antibiotics over the previous 2 weeks leading up to the study.
• Subjects less than 3 years of age

Sponsors & Collaborators

• metaX Institut fuer Diatetik GmbH: lead
• Birmingham Women's and Children's NHS Foundation Trust: collaborator

Study Sites (1)

Birmingham Children´s Hospital

Birmingham, England, B4 6NH, United Kingdom

Location

MeSH Terms

Conditions

Phenylketonurias; 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, Professor

  Birmingham Women's and Children's NHS Foundation Trust

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Bernhard Hoffmann, PhD

CONTACT

+49 6031 166 72 71; behoffmann@metax.org

Yvonne Muecke, PhD

CONTACT

+49 6031 166 72 79; yvonne.muecke@metax.org

Study Design

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

Model Details: open-label, observational

Study Record Dates

• First Submitted: August 31, 2026
• First Posted: September 3, 2026
• Study Start: October 1, 2026
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): August 1, 2027
• Last Updated: September 3, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)