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

NCT ID: NCT03911089

Last Updated: 2021-12-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

3 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-05-28

Study Completion Date

2019-11-30

Brief Summary

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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

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Conditions

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Urea Cycle Disorder Inborn Errors of Metabolism

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Open label

UCD Anamix Infant

Group Type OTHER

UCD Anamix Infant

Intervention Type 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.

Interventions

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UCD Anamix Infant

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.

Intervention Type OTHER

Eligibility Criteria

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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
Maximum Eligible Age

30 Days

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Nutricia Research

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Children's hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Countries

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United States

Other Identifiers

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MPR15IN89539

Identifier Type: -

Identifier Source: org_study_id