The purpose of this prospective, observational study is to evaluate the tolerability and acceptability of Zero minis, a range of protein substitute tablets for use in the dietary management of children with either TYROSINAEMIA Type I, II, III or ALKAPTONURIA, HOMOCYSTINURIA, or MAPLE SYRUP URINE DISEASE (MSUD) over the age of 7 years.
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.
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.
15 children out of a patient pool with proven tyrosinaemia type I, II, III or alkaptonuria, homocystinuria, or MSUD will be recruited from two specialist metabolic centres.
Case-control
Prospective
Zero minis (range of protein substitutes in tablet form)
Dietary supplementIntervention: range of protein substitute tablets. Subjects who currently take a protein substitute for the dietary management of either tyrosinaemias or alkaptonuria, homocystinuria, or MSUD will be recruited. Subjects will take the study product for 7 days. Daily questionnaires will be completed (ease of preparation,administration; any problems or gastrointestinal effects). Subjects will replace some or their entire usual protein substitute with the new product suitable for their diagnosed rare metabolic disease. The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute. Additional questions at the beginning and end of the study will record information on organoleptic properties, presentation and packaging of the product. Routine weekly blood samples will be collected and analysed for amino acids typically for the metabolic control of the individual specified metabolic disorders as is usual clinical practice.
Daily compliance
Compliance, with currently prescribed protein substitute will be assessed at the beginning of the 7-days trial. Usage and compliance with the study product will subsequently assessed daily from days 1-7 using standardised questionnaires, where patients document the the amount of consumed study product vs the prescribed doses.
Treatment-Emergent tolerability
Gastro-intestinal side effects (including diarrhoea, constipation, bloating or abdominal distension, nausea, vomiting, burping, flatulence or regurgitation and abdominal discomfort or pain) will be assessed daily via questionnaire using the scale (none today, mild, moderate or severe / really troublesome).
Patient Acceptability
Acceptability (ease of use and palatability) will be assessed via questionnaire daily during the study period and at the end of the study via questionnaire .
Metabolic control
Weekly routine blood samples will be collected and analysed for specific amino acids typically for the metabolic control of the individual specified metabolic disorders as is usual clinical practice.
Incidence of study product emergent events.
All adverse events will be recorded throughout the study (as and when they occur). There is no specific time frame as the frequency of adverse events can not be predicted. But as the study product is already commercially available occurence of any adverse event is unlikely.
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metaX Institut fuer Diatetik GmbH
Lead sponsor
Birmingham Women's and Children's NHS Foundation Trust
Collaborator
Great Ormond Street Hospital for Children NHS Foundation Trust
Collaborator
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