Market Research - Acceptability Study for a New PKU Protein Substitute

• Sponsor: Dr. Schär AG / SPA (Industry)
• Overall Status: Completed
• CT.gov ID: NCT04318509
• Collaborator: (none)
• Enrollment: 8
• Locations: 2
• Arm: 1
• Actual Duration (Months): 3.4
• Patients Per Site: 4
• Patients Per Site Per Month: 1.2

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
PKU; Phenylketonurias; Inborn Errors of Metabolism; Metabolic Disease	Dietary Supplement: GMPOWER	N/A

Study Design

• Study Type: Interventional
• Actual Enrollment: 8 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Other
• Official Title: Market Research - Acceptability Study for a New PKU Glycomacropeptide Based Protein Substitute (Dr Schär- Mevalia Amino Acids) Mevalia PKU GMPower - Vanilla
• Actual Study Start Date: Feb 4, 2019
• Actual Primary Completion Date: May 19, 2019
• Actual Study Completion Date: May 19, 2019

Arms and Interventions

Arm	Intervention/Treatment
Experimental: PKU GMPOWER (casein) glycomacropeptide protein substitute for the dietary management of PKU from the age of 3 years	Dietary Supplement: GMPOWER; (casein) glycomacropeptide protein substitute for the dietary management of PKU from the age of 3 years

Outcome Measures

Primary Outcome Measures

1. Gastrointestinal tolerance [7 days]

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

2. Product compliance [7 days]

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

3. Product palatability [7 days]

   Questionnaire data captured to evaluate taste

4. Product acceptability [7 days]

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

Eligibility Criteria

Criteria

• Ages Eligible for Study: 3 Years to 16 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• 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).

Exclusion Criteria:

• 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Birmingham Children's Hospital	Birmingham		United Kingdom	B4 6NH
2	Bristol Royal Hospital for Children	Bristol		United Kingdom	BS2 8BJ

Sponsors and Collaborators

• Dr. Schär AG / SPA

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Dr. Schär AG / SPA
• ClinicalTrials.gov Identifier: NCT04318509
• Other Study ID Numbers: GMPower1
• First Posted: Mar 24, 2020
• Last Update Posted: Mar 24, 2020
• Last Verified: Mar 1, 2020

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Phenylketonurias; Metabolism, Inborn Errors; Metabolic Diseases

Study Results

No Results Posted as of Mar 24, 2020