Ignite Creation Date:
2025-12-24 @ 3:01 PM
Ignite Modification Date:
2025-12-26 @ 8:37 PM
Study NCT ID:
NCT04196959
Status:
COMPLETED
Last Update Posted:
2024-02-16
First Post:
2019-12-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Evaluation of TYR Sphere
Sponsor:
Vitaflo International, Ltd
Organization:
Study Overview
Official Title:
An Acceptability Study to Evaluate the Adherence, Tolerance and Metabolic Control of Patients With Tyrosinaemia, When Using TYR Sphere (a Food for Special Medical Purposes (FSMP)) as Part of Dietary Management.
Status:
COMPLETED
Status Verified Date:
2024-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
For 28 days, 20 patients with tyrosinaemia will take TYR sphere as part of the usual dietary management of their condition. Patients will record their adherence to recommended intakes each day, gastrointestinal symptoms (if any) experienced each day of weeks one and four, and the palatability of the product at the end of the trial. Metabolic control will be evaluated by dried blood spots collected during the Baseline and End of Study visits, and weekly during the 28 days of the study itself.
Trial data will be used in an application to make TYR sphere available via the NHS.
Trial data will be used in an application to make TYR sphere available via the NHS.
Detailed Description:
TYR sphere is a glycomacropeptide (GMP) based protein substitute for patients with tyrosinaemia.
The Evaluation of TYR sphere study has been designed to generate data required by the United Kingdom's Advisory Committee on Borderline Substances (ACBS), the national regulator for Foods for Special Medical Purposes (FSMP).
The ACBS specify that before a FSMP can be routinely prescribed by the NHS, its acceptability must first be evaluated by a clinical trial in patients for whom it is ultimately designed. The ACBS define acceptability as comprising patient evaluations of the product's palatability, data on their adherence to recommended intakes and their gastrointestinal tolerance of it. TYR sphere is designed for patients of three years of age and upwards. This has informed the trial's age-related inclusion criteria.
Using a mobile phone application, or paper diary form, patients will record their intake of TYR sphere along with the recommended amount on each of the 28 study days. Gastrointestinal tolerance will be recorded each day during weeks one and four, while palatability of TYR sphere will be recorded at the end of the 28 days, or immediately on discontinuing TYR sphere if the patient stops the trial early. An evaluation of the patient's previous protein substitute and usual GI symptoms are recorded on day one, before the first intake of TYR sphere.
Metabolic control, measured via dried blood spots taken at both visits, and weekly during the 28 days study period, is another primary outcome measure alongside acceptability.
The trial's secondary endpoint is evaluations of growth. Height and weight will be recorded at the Baseline and End of Study visits.
If it is deemed appropriate by the investigator, patients may continue to take the product at the end of the Evaluation Period as part of Care After Research. This continued supply would be given free of charge by the sponsor up until the product is available via a regular prescription, having completed the registration process with the ACBS.
The Evaluation of TYR sphere study has been designed to generate data required by the United Kingdom's Advisory Committee on Borderline Substances (ACBS), the national regulator for Foods for Special Medical Purposes (FSMP).
The ACBS specify that before a FSMP can be routinely prescribed by the NHS, its acceptability must first be evaluated by a clinical trial in patients for whom it is ultimately designed. The ACBS define acceptability as comprising patient evaluations of the product's palatability, data on their adherence to recommended intakes and their gastrointestinal tolerance of it. TYR sphere is designed for patients of three years of age and upwards. This has informed the trial's age-related inclusion criteria.
Using a mobile phone application, or paper diary form, patients will record their intake of TYR sphere along with the recommended amount on each of the 28 study days. Gastrointestinal tolerance will be recorded each day during weeks one and four, while palatability of TYR sphere will be recorded at the end of the 28 days, or immediately on discontinuing TYR sphere if the patient stops the trial early. An evaluation of the patient's previous protein substitute and usual GI symptoms are recorded on day one, before the first intake of TYR sphere.
Metabolic control, measured via dried blood spots taken at both visits, and weekly during the 28 days study period, is another primary outcome measure alongside acceptability.
The trial's secondary endpoint is evaluations of growth. Height and weight will be recorded at the Baseline and End of Study visits.
If it is deemed appropriate by the investigator, patients may continue to take the product at the end of the Evaluation Period as part of Care After Research. This continued supply would be given free of charge by the sponsor up until the product is available via a regular prescription, having completed the registration process with the ACBS.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: