Market Research - Acceptability Study for a New PKU Protein Substitute

Dr. Schär

Status: Completed

Conditions: Phenylketonurias; Inborn Errors of Metabolism; Metabolic Disease; PKU

Treatments: Dietary Supplement: GMPOWER

Study type: Interventional

Funder types: Industry

Identifiers

NCT04318509

GMPower1

Details and patient eligibility

About

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

Enrollment: 8 patients

Sex: All

Ages: 3 to 16 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Other

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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8 participants in 1 patient group

PKU GMPOWER

Experimental group

Description:

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

Treatment:

Dietary Supplement: GMPOWER