Status
Conditions
Treatments
Study type
Funder types
Identifiers
About
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.
Full 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.
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
Exclusion criteria
N.B.: Women who become pregnant during the study will no longer be able to participate and will be withdrawn.
Primary purpose
Allocation
Interventional model
Masking
14 participants in 1 patient group
Loading...
Central trial contact
Kirsten Imbert; Clinical Trials Vitaflo
Data sourced from clinicaltrials.gov
Clinical trials
Research sites
Resources
Legal