Market Research - Acceptability Study for a New PKU Protein Substitute

NCT04318509 | Completed

• 1 other identifier: GMPower1
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 2

Brief Summary

The aim of this study is to demonstrate that a new protein substitute is acceptable and well tolerated in children with PKU.

Conditions

PKU; Phenylketonurias; Inborn Errors of Metabolism; Metabolic Disease

Outcome Measures

Primary Outcomes (4)

• Gastrointestinal tolerance

  Questionnaire detailing any GI symptoms, severity and change from usual • The collection of daily data about the gastro-intestinal tolerance of the protein substitute.

  7 days

• Product compliance

  Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.

  7 days

• Product palatability

  Questionnaire data captured to evaluate taste

  7 days

• Product acceptability

  Brief tick-box questionnaire on overall liking and acceptability of product

  7 days

Study Arms (1)

PKU GMPOWER

EXPERIMENTAL

(casein) glycomacropeptide protein substitute for the dietary management of PKU from the age of 3 years

Dietary Supplement: GMPOWER

Interventions

GMPOWER DIETARY_SUPPLEMENT

(casein) glycomacropeptide protein substitute for the dietary management of PKU from the age of 3 years

PKU GMPOWER

Eligibility Criteria

• Age: 3 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Diagnosis of PKU or PKU variant requiring a protein substitute.
• Subjects who were already taking a phenylalanine-free/low phenylalanine protein substitute and were willing to try the study product for 7 days.
• Patients aged 3 years of age to adulthood.
• Written informed consent obtained from participant or parental caregiver (as appropriate).

You may not qualify if:

• Presence of serious concurrent illness
• Investigator'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 patients having taken antibiotics over the previous 2 weeks leading up to the study.
• Patients less than 3 years of age.

Sponsors & Collaborators

• Dr. Schär AG / SPA: lead

Study Sites (2)

Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom

Location

Bristol Royal Hospital for Children

Bristol, BS2 8BJ, United Kingdom

Location

MeSH Terms

Conditions

Phenylketonurias; Metabolism, Inborn Errors; Metabolic Diseases

Condition Hierarchy (Ancestors)

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

Study Design

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

Study Record Dates

• First Submitted: March 20, 2020
• First Posted: March 24, 2020
• Study Start: February 4, 2019
• Primary Completion: May 19, 2019
• Study Completion: May 19, 2019
• Last Updated: March 24, 2020

Record last verified: 2020-03

Data Sharing

• IPD Sharing: Will not share

Locations

GB (2)