GMP Case Studies of Tolerance, Safety and Acceptability in PKU and TYR

NCT05062226 | Completed

• 1 other identifier: GMP2020
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 1

Brief Summary

Three new protein substitutes have been developed to help with compliance in PKU and TYR patients, which are powdered protein substitutes, low in Phe and, low in Phe and Tyr respectively, with a mixed balance of glycomacropeptide (GMP), essential and non-essential amino acids, carbohydrates, fats and micronutrients for the dietary management of PKU and TYR. This series of case-studies will evaluate the acceptability, compliance, gastrointestinal tolerance and safety of the three GMP-based products for PKU and TYR in 45 patients with PKU and TYR, in both adults and children over 3 years (15 patients per product). The case study will last 29 days in total, including a 1-day baseline period followed by a 28-day intervention period. The case studies will be conducted across multiple specialist metabolic centres in the UK, to meet the UK ACBS and GMS requirements for acceptability studies. A series of case studies is undertaken due to the rarity of these conditions and the difficulty in recruiting these patients to trials.

Conditions

PKU; Tyrosinemias

Keywords

PKU; TYR

Outcome Measures

Primary Outcomes (1)

• Acceptability

  Acceptability of the patients' usual protein substitute and the study product assessed by the Dietitian with a series of questions posed to the patient and/or parent/caregiver on on all attributes which included appearance, smell, taste, texture (mouthfeel), ease of mixing, ease of taking, after taste and smell of breath after taking, assessed on a 5-point likert scale (Great; Good; OK; Bad; Terrible).

  Baseline (Day 1) - End of case studies (Day 29)

Secondary Outcomes (3)

• Compliance

  Baseline (Day 1) - End of case studies (Day 29)

• Gastro-intestinal (GI) tolerance

  Baseline (Day 1) - End of case studies (Day 29)

• Anthropometry

  Baseline (Day 1) - End of case studies (Day 29)

Study Arms (1)

GMP-based products

EXPERIMENTAL

GMP-based nutritional protein substitutes for the dietary management of PKU and TYR

Dietary Supplement: PKU/TYR GMP Protein Substitute

Interventions

PKU/TYR GMP Protein Substitute DIETARY_SUPPLEMENT

After a 1-day baseline period during which patients will consume their usual protein substitute, each patient will receive one the case study product for 4 weeks (28 days).

GMP-based products

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:

• Male or female
• Over 3 years of age
• Diagnosed with classical or variant type phenylketonuria, or tyrosinaemia (as appropriate)
• Have been compliant in taking at least one protein substitute, providing at least 10g protein equivalents, for at least 1 month prior to trial commencement
• Have a prescribed daily Phe or Phe and Tyr allowance for PKU or TYR respectively
• Written or electronic informed consent from patient, and/or from parent/caregiver if applicable

You may not qualify if:

• Currently prescribed Sapropterin or similar tetrahydrobiopterin based medication
• Pregnant or lactating
• Requiring parenteral nutrition
• Major hepatic or renal dysfunction
• Participation in other studies within 1 month prior to entry of this study
• Allergy to any of the study product ingredients, including milk and soy
• Investigator concern around willingness/ability of patient or parent/caregiver to comply with protocol requirements

Sponsors & Collaborators

• Nutricia UK Ltd: lead

Study Sites (1)

Bristol University Hospitals

Bristol, United Kingdom

Location

MeSH Terms

Conditions

Tyrosinemias

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

• Rebecca Stratton

  Nutricia UK Ltd

  STUDY CHAIR

Study Design

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

Model Details: 15 patients per product will be included in the case studies for a total of 45 patients.

Study Record Dates

• First Submitted: September 6, 2021
• First Posted: September 30, 2021
• Study Start: September 16, 2020
• Primary Completion: August 30, 2023
• Study Completion: November 24, 2023
• Last Updated: November 27, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)