Skip to main content
OpenTrials
Completed

NCT Number: NCT03911089

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Urea Cycle Disorder Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Inborn Errors of Metabolism Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Urea Cycle Disorders, Inborn

Age range

Up to 30 day

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Children's hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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.

Primary outcomes

  1. Growth: weight

    Time frame: 16 weeks

    weight [kg]

  2. Growth: length

    Time frame: 16 weeks

    length [cm]

  3. Growth: head circumference

    Time frame: 16 weeks

    head circumference [cm]

Secondary outcomes

  1. GI symptoms [absent/mild/moderate/severe]

    Time frame: 16 weeks

    GI symptoms for the following symptoms:

    • Vomiting (>1 tablespoon/15ml)
    • Abdominal distension
    • Burping
    • Flatulence
    • Diarrhoa
    • Constipation
    • Colic (cramps)
    • Regurgitation (<1 tablespoon/15ml)

  2. Stool frequency

    Time frame: 16 weeks

    stool frequency [# times/day]

  3. Stool consistency

    Time frame: 16 weeks

    stool consistency: quantification score: [Watery; Soft, puddinglike; Soft, formed; Dry, formed; Dry, hard pellets]

  4. Compliance: product intake

    Time frame: 16 weeks

    Intake of medical food compared to amount prescribed [ volume: ml per day]

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

    Time frame: 16 weeks

    Protein, amino acids and nutrient levels in blood [g/dL] or [umol/L]

Other outcomes

  1. Safety and tolerability: (S)AE

    Time frame: 16 weeks

    Occurence of (Serious) Adverse Events

Sponsors and collaborators

Lead sponsor

Nutricia Research

Industry

Registry information

Official study 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

Important dates

Study start
2013
Primary completion
2019
Study completion
2019
First posted
Apr 10, 2019
Registry last updated
Dec 9, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.