Evaluation of TYR Sphere

NCT04196959 | Completed

• 1 other identifier: MCT-TYRS-2019-03-04
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 2

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.

Conditions

Tyrosinemias; Alkaptonuria; Tyrosinemia, Type I; Tyrosinemia, Type II; Tyrosinemia, Type III

Keywords

Tyrosinaemia; AKU; Food for Special Medical Purposes

Outcome Measures

Primary Outcomes (6)

• Adherence to prescribed intake of TYR sphere

  Patient reported data on actual compared to prescribed intakes of TYR sphere

  Days 1 to 28

• Patient record of gastrointestinal tolerance, week 1

  Patient reported data on any gastrointestinal symptoms experienced

  Days 1 - 7

• Patient record of gastrointestinal tolerance, week 4

  Patient reported data on any gastrointestinal symptoms experienced

  Days 21 - 28

• Patient assessment of palatability of TYR sphere

  Patient reported data on the taste, texture, appearance, smell and ease of use of TYR sphere at the end of the trial period.

  Day 28

• Change in metabolic control (tyrosine) over study period

  Tyrosine levels obtained from dried blood spots

  One at Baseline Visit (Day 1) one per week during Evaluation Period (Days 1 - 28) and one at End of Study visit (Day 28)

• Change in metabolic control (phenylalanine) over study period

  Phenylalanine levels obtained from dried blood spots

  One at Baseline Visit (Day 1) one per week during Evaluation Period (Days 1 - 28) and one at End of Study visit (Day 28)

Secondary Outcomes (2)

• Change in height

  Baseline Visit (Day 1) and End of Study Visit (Day 28)

• Change in weight

  Baseline Visit (Day 1) and End of Study Visit (Day 28)

Study Arms (1)

Single Arm

EXPERIMENTAL

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

Dietary Supplement: TYR sphere

Interventions

TYR sphere DIETARY_SUPPLEMENT

TYR sphere is a powdered protein substitute for patients with tyrosinaemia.

Single Arm

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:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Vitaflo International, Ltd: lead

Study Sites (2)

National Alkaptonuria Centre, Royal Liverpool University Hospital

Liverpool, Merseyside, L7 8XP, United Kingdom

Location

Birmingham Children's Hospital

Birmingham, West Midlands, B4 6NH, United Kingdom

Location

MeSH Terms

Conditions

Tyrosinemias; Alkaptonuria

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

  Anita.MacDonald@NHS.Net

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Masking Details: Non-blinded.
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single group, open label.

Study Record Dates

• First Submitted: December 6, 2019
• First Posted: December 12, 2019
• Study Start: October 1, 2019
• Primary Completion: August 1, 2020
• Study Completion: August 1, 2020
• Last Updated: February 16, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

GB (2)