Evaluation of TYR Sphere

Brief Title

Evaluation of TYR Sphere

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.

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.
    

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.
    


Study Type

Interventional


Primary Outcome

Adherence to prescribed intake of TYR sphere

Secondary Outcome

 Change in height

Condition

Tyrosinemias

Intervention

TYR sphere

Study Arms / Comparison Groups

 Single Arm
Description:  All patients will receive TYR sphere, a Food for Special Medical Purposes, as part of thier restricted diet for 28 consecutive days.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Dietary Supplement

Estimated Enrollment

18

Start Date

October 1, 2019

Completion Date

December 18, 2020

Primary Completion Date

August 1, 2020

Eligibility Criteria

        Inclusion Criteria:

          -  Diagnosed with tyrosinaemia.

          -  Aged ≥ 3 years.

          -  In the opinion of the Investigator, can comply with the study protocol and take at
             least one sachet of the study product per day.

          -  Willingly given, written, informed consent from patient or parent/guardian.

          -  Willingly given, written assent (if appropriate).

        Exclusion Criteria:

          -  Individuals who are allergic to milk, fish and soya (these allergens are inherent in
             the study product ingredients).

          -  Use of additional macro/micronutrient supplements during the study period, unless
             clinically indicated and prescribed by the Investigator (must be recorded in patient
             case report form CRF).

          -  Women who are pregnant / breastfeeding at the start of the study or planning to become
             pregnant during the study period.

          -  Individuals who, in the opinion of the investigator, are unable to comply with the
             requirements of the protocol.

          -  Any co-morbidity, which, in the opinion of the Investigator, would preclude
             participation in the study.

          -  Patients who are currently participating in, plan to participate in, or have
             participated in an interventional investigational drug, food or medical device trial
             within 30 days prior to screening visit.
      

Gender

All

Ages

3 Years - N/A

Accepts Healthy Volunteers

No

Contacts

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Location Countries

United Kingdom

Location Countries

United Kingdom

Administrative Informations


NCT ID

NCT04196959

Organization ID

MCT-TYRS-2019-03-04


Responsible Party

Sponsor

Study Sponsor

Vitaflo International, Ltd


Study Sponsor

, , 


Verification Date

December 2020