A Collection of Case Studies in Infants With UCD to Evalu... | NCT03911089

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

NCT03911089

Sponsor

Nutricia Research

Status

Completed

Last Update Posted

Dec 9, 2021Actual

Enrollment

3Actual

Phase

Not Applicable

Conditions

Urea Cycle Disorder
Inborn Errors of Metabolism

Interventions

UCD Anamix Infant

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Dec 2021

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

May 28, 2013Actual

Primary Completion Date

Nov 2019Actual

Completion Date

Nov 2019Actual

First Submitted Date

Aug 28, 2018

First Submission Date that Met QC Criteria

Apr 9, 2019

First Posted Date

Apr 10, 2019Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 8, 2021

Last Update Posted Date

Dec 9, 2021Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

3Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Open label	Other	UCD Anamix Infant	Other: UCD Anamix Infant

Interventions

Name	Type	Description	Arm Group Labels	Other Names
UCD Anamix Infant	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.

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Growth: weight	weight [kg]	16 weeks
Growth: length	length [cm]	16 weeks
Growth: head circumference	head circumference [cm]	16 weeks

Secondary Outcomes

Measure	Description	Time Frame
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)	16 weeks
Stool frequency

Other Outcomes

Measure	Description	Time Frame
Safety and tolerability: (S)AE	Occurence of (Serious) Adverse Events	16 weeks

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

30 Days

Standard Ages

Child

Study Population

Not provided

Sampling Method

Not provided

ID	Term
D056806	Urea Cycle Disorders, Inborn
D008661	Metabolism, Inborn Errors

ID	Term
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D001927	Brain Diseases
D002493	Central Nervous System Diseases