Urea Cycle DisordersNCT01002469
Study to Evaluate 13 C Isotope Ratio Measurement for Urea Cycle Capacity Assessment
A study testing sodium [1-13C] acetate in people with Urea Cycle Disorders, run by Cytonet GmbH & Co. KG at 1 site in Germany.
Trial summary
In this short-term study a method for the evaluation of the metabolic competency of the urea cycle in vivo will be assessed. In order to monitor the efficacy of new treatment options for patients with urea cycle disorders and to monitor the severity of the disease, a reliable and safe quantitative method for the… Read more about this study
- Who is it for?
- Ages 18 to 50 yearsHealthy volunteers can take part
- Conditions the study is for
- Urea Cycle Disorders
- What is being tested?
- sodium [1-13C] acetate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteGermany
- Study dates
- Sep 2009to Dec 2009
- 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 18 to 50 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Group 1 + 2: Healthy subjects
- Group 3: Healthy asymptomatic subjects genetically disposed to urea cycle disorders
- All: age 18 to 50 years (adult)
- Written informed consent
Who cannot join
- Presence of acute infection
- Participation in other clinical trials within 30 days prior to inclusion
- Severe coagulopathy
- Severe systemic or chronic disease
- Pregnancy or lactation
Trial locations and contacts
1 site in 1 country.
Germany1 site
CRS Clinical Research Services Mönchengladbach GmbH
Mönchengladbach, 41061
About this study
Official title: Pilot Feasibility Study in Healthy Subjects and Healthy Asymptomatic Subjects Genetically Disposed to Urea Cycle Disorders to Evaluate the Use of 13 C Isotope Ratio Measurement
In this short-term study a method for the evaluation of the metabolic competency of the urea cycle in vivo will be assessed. In order to monitor the efficacy of new treatment options for patients with urea cycle disorders and to monitor the severity of the disease, a reliable and safe quantitative method for the measurement of the urea cycle flux is required.
Urea synthesis will be evaluated by administering sodium [1-13C]-acetate and measuring subsequent incorporation of [13C] label from Na-acetate into urea in healthy volunteers and asymptomatic subjects genetically disposed to urea cycle disorders.
- Conditions
- Urea Cycle Disorders
- Being tested
- sodium [1-13C] acetate
- Keywords
- isotope ratio measurement, urea cycle, 13C acetate, Healthy adults
- Medical subject headings
- Urea Cycle Disorders, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sodium [1-13C] acetateOther
| Study group | Role | What they receive |
|---|---|---|
| sodium [1-13C] acetate | Experimental | Not described Receives: Other: sodium [1-13C] acetate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Increase of [13C]urea AUC in plasma following oral administration of [13C ]-sodium acetate over baseline
Secondary outcomes (1)
Cmax of absolute [13C]urea in plasma
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2009 (estimated)
- Study startSep 2009
- Primary completionNov 2009
- Study completionDec 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Urea Cycle DisordersStudies of the same condition, open and under way.
- 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
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cytonet GmbH & Co. KG
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Doris Neuenhofer, MD, Principal investigator, CRS Clinical Research Services Mönchengladbach GmbH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01002469
- Sponsor's protocol ID
- CCD07
- Status last verified
- Jan 2010
- First submitted
- 26 Oct 2009
- Last updated
- 15 Jan 2010
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jan 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Feasibility Study in Healthy Subjects and Healthy Asymptomatic Subjects Genetically Disposed to Urea Cycle Disorders to Evaluate the Use of 13 C Isotope Ratio Measurement
- ClinicalTrials.gov ID
- NCT01002469
- Acronym / sponsor ID
- CCD07
- Conditions
- Urea Cycle Disorders
- Keywords
- isotope ratio measurement, urea cycle, 13C acetate, Healthy adults
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Cytonet GmbH & Co. KGIndustry (pharma, biotech, devices)
- Study start
- Sep 2009
- Primary completion
- Nov 2009 (actual)
- Study completion
- Dec 2009 (actual)
- First posted
- 27 Oct 2009 (estimated)
- 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.
In this short-term study a method for the evaluation of the metabolic competency of the urea cycle in vivo will be assessed. In order to monitor the efficacy of new treatment options for patients with urea cycle disorders and to monitor the severity of the disease, a reliable and safe quantitative method for the measurement of the urea cycle flux is required.
Urea synthesis will be evaluated by administering sodium [1-13C]-acetate and measuring subsequent incorporation of [13C] label from Na-acetate into urea in healthy volunteers and asymptomatic subjects genetically disposed to urea cycle disorders.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| sodium [1-13C] acetate | Experimental | Other: sodium [1-13C] acetate |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Group 1 + 2: Healthy subjects
- Group 3: Healthy asymptomatic subjects genetically disposed to urea cycle disorders
- All: age 18 to 50 years (adult)
- Written informed consent
Key exclusion criteria (5 listed)
- Presence of acute infection
- Participation in other clinical trials within 30 days prior to inclusion
- Severe coagulopathy
- Severe systemic or chronic disease
- Pregnancy or lactation
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 |
|---|---|---|
| sodium [1-13C] acetate | Other | sodium [1-13C] acetate |
Primary outcome
Increase of [13C]urea AUC in plasma following oral administration of [13C ]-sodium acetate over baseline
1 secondary outcome
Cmax of absolute [13C]urea in plasma
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 15 Jan 2010.
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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.
Doris Neuenhofer, MD principal investigator
CRS Clinical Research Services Mönchengladbach GmbH
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing sodium [1-13C] acetate in people with Urea Cycle Disorders, run by Cytonet GmbH & Co. KG at 1 site in Germany.
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 4 inclusion and 5 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 15 Jan 2010. 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 Germany. 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?
Increase of [13C]urea AUC in plasma following oral administration of [13C ]-sodium acetate over baseline. 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 15 Jan 2010.