Evaluation of PKU Sphere

Vitaflo

Status: Completed

Conditions: Phenylketonurias

Treatments: Dietary Supplement: PKU Sphere

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT03419819

SCT-PKUS-2016-11-28

Details and patient eligibility

About

A prospective, open-label study of PKU Sphere in patients with PKU following a phenylalanine restricted therapeutic diet.

Full description

The rationale for this study is to assess acceptability, adherence and metabolic control in individuals with PKU consuming PKU Sphere, a GMP-based medical food.

The sponsor developed PKU Sphere in response to growing interest in GMP medical foods for the purpose of improving adherence and quality of life by offering an alternative to amino acid based medical foods. PKU Sphere is a powdered, low phenylalanine medical food containing a balanced mix of casein glycomacropeptide (GMP) isolate, essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals and the long chain polyunsaturated fatty acid (LCP); docosahexaenoic acid (DHA). PKU Sphere has been designed for use in the dietary management of Phenylketonuria. It is available in two flavors, Red Berry and Vanilla, packaged in individual serving sachets of 35g and containing 20g protein equivalent (PE).

Enrollment: 28 patients

Sex: All

Ages: 3+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosis of PKU, identified by newborn screening (NBS) and treated since birth.
• Ages 3 years and above (this includes pregnant women who will be assessed on an individual basis by the Co-Principal Investigators).
• Currently or previously consuming a low phenylalanine diet supplemented with a medical food designed for the dietary management of PKU.
• English as primary language.
• Willingly given, written, informed consent from the participant, 18 or more years, or from the parent/caregiver for participants < 18 years.
• Willingly given, written assent (if appropriate) for those < 18 years.

Exclusion criteria

• Currently consuming 100% of the medical food component of the diet as PKU sphere.
• An inability, in the opinion of the investigator, to comply with the requirements of the protocol.
• Any other type of inherited metabolic disease.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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28 participants in 1 patient group

PKU Sphere

Experimental group

Description:

Phase 1: 1 week To evaluate the acceptability of PKU Sphere during a short-term (1 week) period. Individuals with PKU will aim to consume a minimum of 30% of the medical food component of the diet as PKU Sphere. The amount will be assessed and advised on an individual basis. Phase 2: 4 weeks To evaluate longer-term acceptability and metabolic control in individuals with PKU consuming an agreed target of 50 - 100% of their medical food component of the diet as PKU Sphere for 4 weeks. Some individuals, particularly young children between the ages of 3 - 6 years, may require a 1 - 3 week build up period to reach target volume which will be assessed on an individual basis.

Treatment:

Dietary Supplement: PKU Sphere