Acceptability/Tolerance of Protein Substitutes in Tablet Form for the Dietary Management of Rare Aminoacidopathies

Launched by METAX INSTITUT FUER DIATETIK GMBH · Mar 1, 2024

Keywords

ClinConnect Summary

This clinical trial is looking at how well children over the age of 7 can tolerate and accept a new type of protein substitute tablet called Zero minis. These tablets are designed to help manage specific rare metabolic disorders, including different types of tyrosinemia, alkaptonuria, homocystinuria, and maple syrup urine disease (MSUD). The goal is to see if these tablets are easy for children to take and if they work well for their dietary needs.

To be eligible for this study, children must already have a diagnosis of one of the mentioned disorders and be currently using a protein substitute that meets their dietary requirements. Parents or caregivers will need to give written consent for their child to participate. Participants will try the Zero minis tablets for 7 days to see how well they tolerate them. It’s important to note that children with serious illnesses or those who have taken antibiotics recently may not be able to join the study. Overall, this trial aims to find a convenient way for children with these conditions to get the protein they need while managing their diets effectively.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Diagnosis of Tyrosinaemia type I, II III or Alkaptonuria requiring a tyrosine- and phenylalanine-free protein substitute.
• • Diagnosis of Homocystinuria requiring a methionine-free, cystine-enriched protein substitute.
• • Diagnosis of MSUD requiring a valine-, leucine- and isoleucine-free protein substitute.
• • Subjects who are already taking a protein substitute for one of the specified rare metabolic disorders and are willing to try the study product for 7 days.
• • Children aged 7 years and over.
• • Written informed consent obtained from parental caregiver.
• Exclusion Criteria:
• • Presence of serious concurrent illness
• • Lead Dietitian's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
• • Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
• • Any children having taken antibiotics over the previous 2 weeks leading up to the study.
• • Children less than 7 years of age.