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

NCT03911089 | Completed

• 1 other identifier: MPR15IN89539
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

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.

Conditions

Urea Cycle Disorder; Inborn Errors of Metabolism

Outcome Measures

Primary Outcomes (3)

• Growth: weight

  weight \[kg\]

  16 weeks

• Growth: length

  length \[cm\]

  16 weeks

• Growth: head circumference

  head circumference \[cm\]

  16 weeks

Secondary Outcomes (5)

• GI symptoms [absent/mild/moderate/severe]

  16 weeks

• Stool frequency

  16 weeks

• Stool consistency

  16 weeks

• Compliance: product intake

  16 weeks

• Protein and iron levels from blood samples (for retrospective study part only if data available)

  16 weeks

Other Outcomes (1)

• Safety and tolerability: (S)AE

  16 weeks

Study Arms (1)

Open label

OTHER

UCD Anamix Infant

Other: UCD Anamix Infant

Interventions

UCD Anamix Infant OTHER

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.

Open label

Eligibility Criteria

• Age: Up to 30 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Nutricia Research: lead

Study Sites (1)

Children's hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Urea Cycle Disorders, Inborn; Metabolism, Inborn Errors

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 28, 2018
• First Posted: April 10, 2019
• Study Start: May 28, 2013
• Primary Completion: November 1, 2019
• Study Completion: November 1, 2019
• Last Updated: December 9, 2021

Record last verified: 2021-12

Locations

US (1)