Clinical trial NCT03911089: A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Urea Cycle Disorder
Inborn Errors of Metabolism
Lead sponsor: Nutricia Research
Sponsor class: INDUSTRY
First posted: 2019-04-10
Enrollment: 3
Sex: ALL
Maximum age (years): 30
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Growth: weight
timeFrame: 16 weeks
measure: Growth: length
timeFrame: 16 weeks
measure: Growth: head circumference
timeFrame: 16 weeks
Secondary outcomes:
measure: GI symptoms [absent/mild/moderate/severe]
timeFrame: 16 weeks
measure: Stool frequency
timeFrame: 16 weeks
measure: Stool consistency
timeFrame: 16 weeks
measure: Compliance: product intake
timeFrame: 16 weeks
measure: Protein and iron levels from blood samples (for retrospective study part only if data available)
timeFrame: 16 weeks
Other outcomes:
measure: Safety and tolerability: (S)AE
timeFrame: 16 weeks
Locations:
city: Philadelphia
country: United States
facility: Children's hospital of Philadelphia
state: Pennsylvania
Study officials:
References: