Inborn Errors of Metabolism, Urea Cycle Disorder
Conditions
Brief summary
Original study: Study aims to enroll 3-5 children with confirmed Urinary Cycle Disorder (UCD). Subjects meeting the inclusion and exclusion criteria are included during the baseline visit. All subjects will receive the investigational product for a period of 16 weeks. At baseline, 2, 4, 8, 12 and 16 weeks the study parameters are assessed. The study amendment aims to collect case studies retrospectively of children who have used UCD Anamix Infant for at least 16 weeks, in countries where UCD Anamix Infant is already available on the market. It is aimed to collect the same study parameters of the original study at preferably the same timepoints.
Interventions
OTHERUCD Anamix Infant
UCD Anamix infant with DHA and ARA is an amino acid based infant medical food containing only the essential amino acids, carbohydrate, fat, vitamins, minerals and trace elements.
Sponsors
Nutricia Research
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 30 Days
Healthy volunteers
No
Inclusion criteria
Prospective study: * Male and female infants diagnosed with UCD, aged from birth to 30 days of age * Parent / primary caregiver is able to give informed consent * Infants who will receive and will be able to take study medical food as part (40-70%) of their protein requirements in the day Retrospective study: * Male and female term infants diagnosed with UCD, that started UCD Anamix Infant when aged from birth to 30 days of age. * Parent / primary caregiver is able to give informed consent * Infants who received UCD Anamix Infant as part of their protein requirements (40-70%) in the day for at least 16 weeks.
Exclusion criteria
* Infants \< 5lb 8oz (\< 2.5 kg) at birth * Infants \<34 weeks of gestation and using a special premature formula at study start * Infants with severe concurrent illness or major congenital malformations, except conditions consequent to urea cycle disorder * Infants with suspected or documented systemic or congenital infections (e.g. human immunodeficiency virus) * Infants with evidence of significant cardiac, respiratory, haematological, gastrointestinal, or other systemic diseases * Infants currently taking cytotoxic drugs * Investigator's uncertainty about the willingness or ability of the parent to comply with the protocol requirements * Participation in any other study involving investigational products concomitantly or prior to entry into the study * An infant of any personnel connected with the study * Infants whose parent / primary caregiver is younger than the legal age of consent
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Growth: head circumference | 16 weeks | head circumference \[cm\] |
| Growth: weight | 16 weeks | weight \[kg\] |
| Growth: length | 16 weeks | length \[cm\] |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Stool consistency | 16 weeks | stool consistency: quantification score: \[Watery; Soft, puddinglike; Soft, formed; Dry, formed; Dry, hard pellets\] |
| Protein and iron levels from blood samples (for retrospective study part only if data available) | 16 weeks | Protein, amino acids and nutrient levels in blood \[g/dL\] or \[umol/L\] |
| Compliance: product intake | 16 weeks | Intake of medical food compared to amount prescribed \[ volume: ml per day\] |
| GI symptoms [absent/mild/moderate/severe] | 16 weeks | GI symptoms for the following symptoms: * Vomiting (\>1 tablespoon/15ml) * Abdominal distension * Burping * Flatulence * Diarrhoa * Constipation * Colic (cramps) * Regurgitation (\<1 tablespoon/15ml) |
| Stool frequency | 16 weeks | stool frequency \[# times/day\] |
Other
| Measure | Time frame | Description |
|---|---|---|
| Safety and tolerability: (S)AE | 16 weeks | Occurence of (Serious) Adverse Events |
Countries
United States
Outcome results
None listed