Hyperammonemic EncephalopathyNCT05754372
Optimalisation of the Treatment of Acute Neonatal Hyperammonaemia
An observational study of Hyperammonemic Encephalopathy, run by University Hospital, Ghent at 1 site in Belgium.
Trial summary
Acute neonatal hyperammonemia is associated with poor neurological outcomes and high mortality. A user-friendly and widely applicable algorithm - based on kinetics - to tailor the treatment of acute neonatal hyperammonemia. Read more about this study
- Who is it for?
- Ages Up to 1 year
- Conditions the study is for
- Hyperammonemic Encephalopathy
- What is being tested?
- user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemia
- How many people
- 10 participants
- Where
- 1 siteBelgium
- Study dates
- 15 Jan 2023to 31 Dec 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 1 year
- All sexes
- No healthy volunteers
- Child
Study population: infants presenting with acute neonatale hyperammonaemia
Who may be able to join
- severe acute neonatal hyperammonaemia for which acute hemodialysis is indicated
- inborn error of metabolism
- below 1 year of age
Who cannot join
- older than 1 year
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Belgium1 site, 1 recruiting
- Recruiting
Ghent University Hospital - Department of Paediatric Nephrology
Ghent, 9000
Evelien Snauwaert, MD PhD, 0032 9 332 5271, evelien.snauwaert@uzgent.be
Sunny Eloot, PhD, sunny.eloot@ugent.be
About this study
Acute neonatal hyperammonemia is associated with poor neurological outcomes and high mortality. A user-friendly and widely applicable algorithm - based on kinetics - to tailor the treatment of acute neonatal hyperammonemia. A single compartmental model was calibrated assuming a distribution volume equal to the patient's total body water (V), as calculated using Wells' formula, and dialyzer clearance as derived from the measured ammonia time-concentration curves during 11 dialysis sessions in four patients (3.2 +/- 0.4 kg). Based on these kinetic simulations, dialysis protocols could be derived for clinical use with different body weights, start concentrations, dialysis machines/dialyzers and dialysis settings (e.g., blood flow QB). By a single measurement of ammonia concentration at the dialyzer inlet and outlet, dialyzer clearance (K) can be calculated as K = QB[(Cinlet - Coutlet)/Cinlet]. The time (T) needed to decrease the ammonia concentration from a predialysis start concentration Cstart to a desired target concentration Ctarget is then equal to T = (-V/K)LN(Ctarget/Cstart). By implementing these formulae in a simple spreadsheet, medical staff can draw an institution-specific flowchart for patient-tailored treatment of hyperammonemia.
The aim of this study is to validate these formula with a prospective study.
- Conditions
- Hyperammonemic Encephalopathy
- Being tested
- user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemia
- Keywords
- hemodialysis, neonate, Hyperammonemic Encephalopathy, inborn error of metbolism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemiaDiagnostic test
user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemia, based on kinetic modelling
| Study group | Role | What they receive |
|---|---|---|
| Hyperammonaemia | Not described Receives: Diagnostic Test: user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemia |
What is being measured
The primary outcome is the main question the study is designed to answer.
serum ammonium levels
serum ammonium levels
Measured: 1 year
Secondary outcomes (1)
Removal of treatment sodium benzoate, L-arginine
serum and dialysate concentration (mg/dL) of sodium benzoate and L-arginine
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2023
- Study start15 Jan 2023
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hyperammonemic EncephalopathyStudies of the same condition, open and under way.
- More trials run by University Hospital, GhentThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Ghent
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05754372
- Sponsor's protocol ID
- BC-10637
- Status last verified
- Apr 2024
- First submitted
- 3 Jan 2022
- Last updated
- 15 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Optimalisation of the Treatment of Acute Neonatal Hyperammonaemia
- ClinicalTrials.gov ID
- NCT05754372
- Acronym / sponsor ID
- BC-10637
- Conditions
- Hyperammonemic Encephalopathy
- Keywords
- hemodialysis, neonate, Hyperammonemic Encephalopathy, inborn error of metbolism
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- University Hospital, GhentUniversity, hospital or other
- Study start
- 15 Jan 2023 (actual)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 3 Mar 2023 (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.
Acute neonatal hyperammonemia is associated with poor neurological outcomes and high mortality. A user-friendly and widely applicable algorithm - based on kinetics - to tailor the treatment of acute neonatal hyperammonemia. A single compartmental model was calibrated assuming a distribution volume equal to the patient's total body water (V), as calculated using Wells' formula, and dialyzer clearance as derived from the measured ammonia time-concentration curves during 11 dialysis sessions in four patients (3.2 +/- 0.4 kg). Based on these kinetic simulations, dialysis protocols could be derived for clinical use with different body weights, start concentrations, dialysis machines/dialyzers and dialysis settings (e.g., blood flow QB). By a single measurement of ammonia concentration at the dialyzer inlet and outlet, dialyzer clearance (K) can be calculated as K = QB[(Cinlet - Coutlet)/Cinlet]. The time (T) needed to decrease the ammonia concentration from a predialysis start concentration Cstart to a desired target concentration Ctarget is then equal to T = (-V/K)LN(Ctarget/Cstart). By implementing these formulae in a simple spreadsheet, medical staff can draw an institution-specific flowchart for patient-tailored treatment of hyperammonemia.
The aim of this study is to validate these formula with a prospective study.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hyperammonaemia | – | Diagnostic Test: user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemia |
Who may qualify
- Ages
- Up to 1 year
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- infants presenting with acute neonatale hyperammonaemia
Key inclusion criteria (3 listed)
- severe acute neonatal hyperammonaemia for which acute hemodialysis is indicated
- inborn error of metabolism
- below 1 year of age
Key exclusion criteria (1 listed)
- older than 1 year
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 |
|---|---|---|
| user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemiauser-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemia, based on kinetic modelling | Diagnostic test | Hyperammonaemia |
Primary outcome
serum ammonium levels
Time frame: 1 year
serum ammonium levels
1 secondary outcome
Removal of treatment sodium benzoate, L-arginine
Time frame: 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2024.
- Individual participant dataSharing planned: No
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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hyperammonemic Encephalopathy, run by University Hospital, Ghent at 1 site in Belgium.
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 3 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 15 Apr 2024. 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 Belgium. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Evelien Snauwaert, MD, PhD (00 32 9 332 69 05, evelien.snauwaert@uzgent.be), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
serum ammonium levels (time frame: 1 year). 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 Apr 2024.