A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD
Primary Purpose
Urea Cycle Disorder, Inborn Errors of Metabolism
Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
UCD Anamix Infant
Sponsored by
About this trial
This is an interventional treatment trial for Urea Cycle Disorder
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
Sites / Locations
- Children's hospital of Philadelphia
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
Open label
Arm Description
UCD Anamix Infant
Outcomes
Primary Outcome Measures
Growth: weight
weight [kg]
Growth: length
length [cm]
Growth: head circumference
head circumference [cm]
Secondary Outcome Measures
GI symptoms [absent/mild/moderate/severe]
GI symptoms for the following symptoms:
Vomiting (>1 tablespoon/15ml)
Abdominal distension
Burping
Flatulence
Diarrhoa
Constipation
Colic (cramps)
Regurgitation (<1 tablespoon/15ml)
Stool frequency
stool frequency [# times/day]
Stool consistency
stool consistency: quantification score: [Watery; Soft, puddinglike; Soft, formed; Dry, formed; Dry, hard pellets]
Compliance: product intake
Intake of medical food compared to amount prescribed [ volume: ml per day]
Protein and iron levels from blood samples (for retrospective study part only if data available)
Protein, amino acids and nutrient levels in blood [g/dL] or [umol/L]
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03911089
Brief Title
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
2. Study Status
Record Verification Date
December 2021
Overall Recruitment Status
Completed
Study Start Date
May 28, 2013 (Actual)
Primary Completion Date
November 2019 (Actual)
Study Completion Date
November 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Nutricia Research
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Urea Cycle Disorder, Inborn Errors of Metabolism
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
3 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Open label
Arm Type
Other
Arm Description
UCD Anamix Infant
Intervention Type
Other
Intervention Name(s)
UCD Anamix Infant
Intervention Description
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 Outcome Measure Information:
Title
Growth: weight
Description
weight [kg]
Time Frame
16 weeks
Title
Growth: length
Description
length [cm]
Time Frame
16 weeks
Title
Growth: head circumference
Description
head circumference [cm]
Time Frame
16 weeks
Secondary Outcome Measure Information:
Title
GI symptoms [absent/mild/moderate/severe]
Description
GI symptoms for the following symptoms:
Vomiting (>1 tablespoon/15ml)
Abdominal distension
Burping
Flatulence
Diarrhoa
Constipation
Colic (cramps)
Regurgitation (<1 tablespoon/15ml)
Time Frame
16 weeks
Title
Stool frequency
Description
stool frequency [# times/day]
Time Frame
16 weeks
Title
Stool consistency
Description
stool consistency: quantification score: [Watery; Soft, puddinglike; Soft, formed; Dry, formed; Dry, hard pellets]
Time Frame
16 weeks
Title
Compliance: product intake
Description
Intake of medical food compared to amount prescribed [ volume: ml per day]
Time Frame
16 weeks
Title
Protein and iron levels from blood samples (for retrospective study part only if data available)
Description
Protein, amino acids and nutrient levels in blood [g/dL] or [umol/L]
Time Frame
16 weeks
Other Pre-specified Outcome Measures:
Title
Safety and tolerability: (S)AE
Description
Occurence of (Serious) Adverse Events
Time Frame
16 weeks
10. Eligibility
Sex
All
Maximum Age & Unit of Time
30 Days
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
Children's hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
12. IPD Sharing Statement
Learn more about this trial
A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD
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