Evaluation of TYR Sphere in France

NCT04761588 | Completed

• 3 other identifiers: TyrSph-2020-10-16SI 21.01.06.741212021-A01263-38
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Tyrosinemia; AKU

Keywords

Tyrosinemia; AKU; Alkaptonuria; Tyr Sphere; FSMP; Food for special medical purposes

Outcome Measures

Primary Outcomes (5)

• Change in adherence to recommended amounts of Tyr sphere

  Patient reported data on actual compared to prescribed intakes of Tyr sphere, assessed by HCPs periodically as per routine practice.

  Recorded daily on days 1-28

• Change in gastrointestinal tolerance

  Patient reported data on any gastrointestinal symptoms experienced, assessed by HCPs periodically as per routine practice.

  Recorded daily on days 1-28

• Patient evaluation of Tyr sphere's palatability

  Patient-reported assessment of Tyr sphere's palatability on a Likert scale: 1 (really didn't like it) to 5 (loved it).

  Day 28, end of acceptability phase

• Change in metabolic control: tyrosine levels

  Tyrosine levels obtained from dried blood spots

  Day 1, week 1, week 2, week 3, week 4, day 28

• Change in metabolic control: phenylalanine levels

  Phenylalanine levels obtained from dried blood spots

  Day 1, week 1, week 2, week 3, week 4, day 28

Secondary Outcomes (4)

• Long term change in growth

  3-year follow-up period

• Long term change in urine succinylacetone level

  3-year follow-up period

• Long term change in pre-albumin level

  3-year follow-up period

• Long term change in serum albumin level

  3-year follow-up period

Study Arms (1)

Tyr sphere

EXPERIMENTAL

All patients to receive Tyr sphere as part of their dietary management for tyrosinaemia or alkaptonuria (AKU).

Dietary Supplement: Tyr sphere

Interventions

Tyr sphere DIETARY_SUPPLEMENT

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.

Tyr sphere

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 evaluation 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.
• N.B.: Women who become pregnant during the study will no longer be able to participate and will be withdrawn.
• 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 (1)

Hopital Necker-Enfants Malades

Paris, France

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

• Jean-Baptiste Arnoux

  Hopital Necker-Enfants Malades

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: February 15, 2021
• First Posted: February 21, 2021
• Study Start: December 1, 2021
• Primary Completion: December 31, 2024
• Study Completion: October 31, 2025
• Last Updated: November 28, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)