Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of Phenylketonuria

NCT04375592 | Completed

• 1 other identifier: XPheminisCT01
• Study Type: observational
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Phenylketonurias; Hyperphenylalaninaemia; Tetrahydrobiopterin Deficiency

Outcome Measures

Primary Outcomes (1)

• Daily compliance

  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

  7 days

Secondary Outcomes (4)

• Treatment-Emergent tolerability

  7 days

• Patient Acceptability

  7 days

• Metabolic control

  2 days

• Incidence of study product emergent events.

  Through study completion an average of 1year

Interventions

Phenylalanine-free protein substitute in tablet form (XPhe minis) DIETARY_SUPPLEMENT

Intervention is a Phenylalanine-free protein substitute in tablet form. Subjects who currently take a concentrated second stage phenylalanine-free protein substitute will be recruited. Subjects will take the study product for 7 days and daily questionnaires will be completed. 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. Daily acceptability and tolerance questionnaires will record information on ease of preparation and administration; how it is taken; and any problems or gastrointestinal effects. Additional questions at the beginning and end of the study will record information on taste, appearance, smell, presentation and packaging of the product. Routine weekly finger prick blood spots will be collected and analysed for phenylalanine and tyrosine as usual.

Eligibility Criteria

• Age: 7 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

10 children with PKU

You may qualify if:

• 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.

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 children having taken antibiotics over the previous 2 weeks leading up to the study.
• Children less than 7 years of age.

Sponsors & Collaborators

• metaX Institut fuer Diatetik GmbH: lead
• Birmingham Children's Hospital: collaborator

Study Sites (1)

Birmingham Children´s Hospital

Birmingham, Wales, 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, Professor

  Birmingham Children´s Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 21, 2020
• First Posted: May 5, 2020
• Study Start: February 1, 2021
• Primary Completion: May 1, 2021
• Study Completion: May 10, 2021
• Last Updated: March 4, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)