Evaluation of PKU Sphere Liquid

NCT05096988 | Completed

• 3 other identifiers: MCT-GMPL-2018-02-2127180120/NW/0273
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 3

Brief Summary

PKU Sphere Liquid is a prospective, open-label, acceptability study to evaluate PKU sphere liquid in up to 15 participants aged 3 and above for the dietary management of PKU over 31 days.

Conditions

Phenylketonurias

Keywords

PKU; Sphere; Liquid

Outcome Measures

Primary Outcomes (3)

• Questionnaire of self-reported adherence to the prescribed amount of study product

  Assessment of participant's adherence to prescribed amount during the eight week intake of the study product

  4 weeks

• Product acceptability rated on a Likert scale by the participant after four week intake

  Assessment of participant's acceptability following four week intake of the study product Scale is from 1 to 5: 1 = Loved it, 5 = Really didn't like it

  4 weeks

• Questionnaire of self-reported changes in gastrointestinal tolerance during four week intake

  Assessment of participant's gastrointestinal tolerance during the four week intake of the study product

  4 weeks

Study Arms (1)

PKU sphere liquid

EXPERIMENTAL

PKU sphere liquid will be prescribed by the study dietitian based on the patient's individual requirements.

Dietary Supplement: PKU sphere liquid

Interventions

PKU sphere liquid DIETARY_SUPPLEMENT

PKU sphere liquid will be prescribed by the study dietitian based on the patient's individual requirements.

PKU sphere liquid

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of PKU requiring a low protein diet and protein substitute.
• Aged three (3) years and over
• Already taking a minimum of one GMP based protein substitute per day or have previously taken one
• Able to comply with the study protocol and take the study product, according to the opinion of the PI.
• Willingly given, written, informed consent from patient or parent/guardian.
• Willingly given, written assent (if appropriate).

You may not qualify if:

• Inability to comply with the study protocol, in the opinion of the investigator.
• Any co-morbidity, which, in the opinion of the investigator, would preclude participation in the study.
• Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary intervention with a low protein diet and protein supplement.
• Unwilling/unable to take a GMP based protein substitute.
• Women who are pregnant / breastfeeding at the start of the study or planning to become pregnant during the study period.
• N.B.: Women who become pregnant unexpectedly during this study will be immediately withdrawn from the study.

Sponsors & Collaborators

• Vitaflo International, Ltd: lead

Study Sites (3)

Royal Victoria Hospital

Belfast, BT12 6BA, United Kingdom

Location

Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom

Location

Northern General Hospital

Sheffield, S5 7AU, 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

  Birmingham Women's and Children's NHS Foundation Trust

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: October 14, 2021
• First Posted: October 27, 2021
• Study Start: November 23, 2021
• Primary Completion: June 21, 2022
• Study Completion: February 28, 2023
• Last Updated: February 16, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

GB (3)