NCT04761588

Evaluation of TYR Sphere in France

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) as Part of Dietary Management.

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: Vitaflo International, Ltd · Industry
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

Summary

10 participants aged 3 years and over with tyrosinaemia or alkaptonuria will be prescribed Tyr sphere following an assessment of their individual needs by their dietitian. All participants will enter a 4-week evaluation period, assessing adherence and gastrointestinal tolerance. Evaluations of Tyr sphere's palatability are made at the end of the evaluation period. Dried blood spots are taken on days 1 and 28 and once per week in between. Participants who continue to take the product at the end of their evaluation period will enter a follow-up period during which metabolic control, anthropometric and nutritional status data will be collected during the yearly standard of care routine visits.

Detailed description

10 participants with tyrosinaemia or alkaptonuria will be invited to participate in this study. Following consent they will be assessed by their dietitian and a recommended amount of Tyr sphere will be prescribed based on their individual needs. Tyr sphere is a food for special medical purposes as defined by Regulation (EU) No 609/2013. It is designed to be prescribed based on its protein content, not its energy content. Visit 1, baseline clinic visit activities: consent, demographics, anthropometric measurements, gastrointestinal history, dietary assessment, metabolic control assessment including collection of dried blood spots for phenylalanine and tyrosine levels, record routinely taken urine succinylacetone, serum albumin and pre-albumin levels, new dietary prescription. Weeks 1 to 4 (patient at home): dried blood spots, adverse events reporting/assessment, daily Tyr sphere adherence diary, daily gastrointestinal symptoms diary (weeks 1 and 4 only), daily intake diary for Nitisinone which is routinely prescribed. There will be a telephone call Visit 2, end of acceptability phase clinic visit activities: anthropometric measurements, review of diaries, review of adverse events, metabolic control assessment including collection of dried blood spots for phenylalanine and tyrosine levels, investigator's assessment of Tyr sphere for the participant and decision on continuing with the prescription. Three-year routine follow-up: for the participants that do continue with the Tyr sphere prescription (in consultation with the investigator), they will enter a follow-up period of three years, during which only routine, standard of care visits and procedures will take place. Data will be collected on anthropometrics, phenylalanine and tyrosine levels from dried blood spots, adverse events, record routinely taken urine succinylacetone, serum albumin and pre-albumin levels.

Conditions

• Tyrosinemia
• AKU

Interventions

Type	Name	Description
DIETARY_SUPPLEMENT	Tyr sphere	Tyr sphere is a powdered, low phenylalanine and tyrosine protein substitute, containing a balanced mix of casein glycomacropeptide (cGMP) isolate, essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals and the long chain polyunsaturated fatty acid (LCP) and docosahexaenoic acid (DHA). It contains sugars and sweetener. The product is designed to be prescribed based on its protein content, not its energy content.

Timeline

• Start date: 2021-12-01
• Primary completion: 2024-12-31
• Completion: 2025-10-31
• First posted: 2021-02-21
• Last updated: 2025-11-28

Locations

1 site across 1 country: France