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

Not Applicable

Completed

• Conditions: Inborn Errors of Metabolism; Urea Cycle Disorder
• Interventions: Other: UCD Anamix Infant

• Registration Number: NCT03911089
• Lead Sponsor: Nutricia Research

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2013-05-28
• Study First Submitted: 2018-08-28
• Study First Submitted QC: 2019-04-09
• Study First Posted: 2019-04-10
• Primary Completion: 2019-11
• Study Completion: 2019-11
• Status Verified: 2021-12
• Last Update Submitted: 2021-12-08
• Last Update Posted: 2021-12-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 3

Inclusion Criteria

Not provided

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open label	UCD Anamix Infant	UCD Anamix Infant

Primary Outcome Measures

Name	Time	Method
Growth: head circumference	16 weeks	head circumference \[cm\]
Growth: weight	16 weeks	weight \[kg\]
Growth: length	16 weeks	length \[cm\]

Secondary Outcome Measures

Name	Time	Method
Stool consistency	16 weeks	stool consistency: quantification score: \[Watery; Soft, puddinglike; Soft, formed; Dry, formed; Dry, hard pellets\]
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 \[# times/day\]
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 \[umol/L\]

Trial Locations

Locations (1)

Children's hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States