Urea Cycle DisorderNCT03911089
A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD
A study testing UCD Anamix Infant in people with Urea Cycle Disorder and Inborn Errors of Metabolism, run by Nutricia Research at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 30 days
- Conditions the study is for
- Urea Cycle Disorder, Inborn Errors of Metabolism
- What is being tested?
- UCD Anamix Infant
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 28 May 2013to Nov 2019
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 30 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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
Show all 10Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
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
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.
- Conditions
- Urea Cycle Disorder, Inborn Errors of Metabolism
- Being tested
- UCD Anamix Infant
- Medical subject headings
- Urea Cycle Disorders, Inborn, Metabolism, Inborn Errors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UCD Anamix InfantOther
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.
| Study group | Role | What they receive |
|---|---|---|
| Open label | Other | UCD Anamix Infant Receives: Other: UCD Anamix Infant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Growth: weight
weight [kg]
Measured: 16 weeks
Growth: length
length [cm]
Measured: 16 weeks
Growth: head circumference
head circumference [cm]
Measured: 16 weeks
Secondary outcomes (5)
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)
Measured: 16 weeks
Stool frequency
stool frequency [# times/day]
Measured: 16 weeks
Stool consistency
stool consistency: quantification score: [Watery; Soft, puddinglike; Soft, formed; Dry, formed; Dry, hard pellets]
Measured: 16 weeks
Compliance: product intake
Intake of medical food compared to amount prescribed [ volume: ml per day]
Measured: 16 weeks
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]
Measured: 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2019
- Study start28 May 2013
- Primary completionNov 2019
- Study completionNov 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Urea Cycle DisorderStudies of the same condition, open and under way.
- More trials run by Nutricia ResearchThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nutricia Research
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03911089
- Sponsor's protocol ID
- MPR15IN89539
- Status last verified
- Dec 2021
- First submitted
- 28 Aug 2018
- Last updated
- 9 Dec 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03911089
- Acronym / sponsor ID
- MPR15IN89539
- Conditions
- Urea Cycle Disorder, Inborn Errors of Metabolism
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Nutricia ResearchIndustry (pharma, biotech, devices)
- Study start
- 28 May 2013 (actual)
- Primary completion
- Nov 2019 (actual)
- Study completion
- Nov 2019 (actual)
- First posted
- 10 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open label | Other | Other: UCD Anamix InfantUCD Anamix Infant |
Who may qualify
- Ages
- Up to 30 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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
- 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
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| UCD Anamix InfantUCD 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. | Other | Open label |
Primary outcome
Growth: weight
Time frame: 16 weeks
weight [kg]
Growth: length
Time frame: 16 weeks
length [cm]
Growth: head circumference
Time frame: 16 weeks
head circumference [cm]
5 secondary outcomes
GI symptoms [absent/mild/moderate/severe]
Time frame: 16 weeks
Stool frequency
Time frame: 16 weeks
Stool consistency
Time frame: 16 weeks
Compliance: product intake
Time frame: 16 weeks
Protein and iron levels from blood samples (for retrospective study part only if data available)
Time frame: 16 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Dec 2021.
Related trials for Urea Cycle Disorder
RecruitingObservational study
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Not yet recruitingObservational study
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
RecruitingObservational study
Abbott Vascular Medical Device Registry
Acute Myocardial Infarction, Restenoses, Coronary
RecruitingPhase 1/2
Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD
Urea Cycle Disorders, Inborn, Urea Cycle Disorders
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing UCD Anamix Infant in people with Urea Cycle Disorder and Inborn Errors of Metabolism, run by Nutricia Research at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Growth: weight (time frame: 16 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Dec 2021.