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

NCT05062226 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 45

Last updated 2023-11-27

No results posted yet for this study

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

Interventions

DIETARY_SUPPLEMENT

PKU/TYR GMP Protein Substitute

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).

Sponsors & Collaborators

  • Nutricia UK Ltd

    lead INDUSTRY

Principal Investigators

  • Rebecca Stratton · Nutricia UK Ltd

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-09-16
Primary Completion
2023-08-30
Completion
2023-11-24

Countries

  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05062226 on ClinicalTrials.gov