Clincosm LogoSign in Get a demo

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

Sponsor
Nutricia Research (Industry)
Overall Status
Completed
CT.gov ID
NCT03911089
Collaborator
(none)
3
Enrollment
1
Location
1
Arm
77.1
Actual Duration (Months)
0
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Other: UCD Anamix Infant
 N/A

Study Design

Study Type:
Interventional
Actual Enrollment :
3 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
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
Actual Study Start Date :
May 28, 2013
Actual Primary Completion Date :
Nov 1, 2019
Actual Study Completion Date :
Nov 1, 2019

Arms and Interventions

Arm Intervention/Treatment
Other: Open label

UCD Anamix Infant

Other: 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.

Outcome Measures

Primary Outcome Measures

  1. Growth: weight [16 weeks]

    weight [kg]

  2. Growth: length [16 weeks]

    length [cm]

  3. Growth: head circumference [16 weeks]

    head circumference [cm]

Secondary Outcome Measures

  1. GI symptoms [absent/mild/moderate/severe] [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 [16 weeks]

    stool frequency [# times/day]

  3. Stool consistency [16 weeks]

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

  4. Compliance: product intake [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) [16 weeks]

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

Other Outcome Measures

  1. Safety and tolerability: (S)AE [16 weeks]

    Occurence of (Serious) Adverse Events

Eligibility Criteria

Criteria

Ages Eligible for Study:
N/A to 30 Days
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Children's hospital of Philadelphia Philadelphia Pennsylvania United States 19104

Sponsors and Collaborators

  • Nutricia Research

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Nutricia Research
ClinicalTrials.gov Identifier:
NCT03911089
Other Study ID Numbers:
  • MPR15IN89539
First Posted:
Apr 10, 2019
Last Update Posted:
Dec 9, 2021
Last Verified:
Dec 1, 2021
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Urea Cycle Disorders, Inborn
Metabolism, Inborn Errors

Study Results

No Results Posted as of Dec 9, 2021