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

Nutricia

Status

Completed

Conditions

Inborn Errors of Metabolism

Urea Cycle Disorder

Treatments

Other: UCD Anamix Infant

Study type

Interventional

Funder types

Industry

Identifiers

NCT03911089

MPR15IN89539

Details and patient eligibility

About

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.

Enrollment

3 patients

Sex

All

Ages

Under 30 days old

Volunteers

No Healthy Volunteers

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