A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD

Inborn Errors of Metabolism Clinical Trial

Official title:

A Collection of Case Studies in Infants With Urea Cycle Disorders (UCD) to Evaluate Infant Growth and the Safety of a New Medical Food for UCD

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03911089
• Other study ID #: MPR15IN89539
• Status: Completed
• Phase: N/A
• Start date: May 28, 2013
• Est. completion date: November 2019

Study information

• Verified date: December 2021
• Source: Nutricia Research
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 3
• Est. completion date: November 2019
• Est. primary completion date: November 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 30 Days

Eligibility

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

Related Conditions & MeSH terms

• Inborn Errors of Metabolism
• Metabolism, Inborn Errors
• Urea Cycle Disorder
• Urea Cycle Disorders, Inborn

Intervention

Other:
• UCD 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.

Locations

Country	Name	City	State
United States	Children's hospital of Philadelphia	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Nutricia Research

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Safety and tolerability: (S)AE	Occurence of (Serious) Adverse Events	16 weeks
Primary	Growth: weight	weight [kg]	16 weeks
Primary	Growth: length	length [cm]	16 weeks
Primary	Growth: head circumference	head circumference [cm]	16 weeks
Secondary	GI symptoms [absent/mild/moderate/severe]	GI symptoms for the following symptoms: Vomiting (>1 tablespoon/15ml); Abdominal distension; Burping; Flatulence; Diarrhoa; Constipation; Colic (cramps); Regurgitation (<1 tablespoon/15ml)	16 weeks
Secondary	Stool frequency	stool frequency [# times/day]	16 weeks
Secondary	Stool consistency	stool consistency: quantification score: [Watery; Soft, puddinglike; Soft, formed; Dry, formed; Dry, hard pellets]	16 weeks
Secondary	Compliance: product intake	Intake of medical food compared to amount prescribed [ volume: ml per day]	16 weeks
Secondary	Protein and iron levels from blood samples (for retrospective study part only if data available)	Protein, amino acids and nutrient levels in blood [g/dL] or [umol/L]	16 weeks