Optimalisation of the Treatment of Acute Neonatal Hyperammonaemia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemia.
- Who it may be relevant to
- Registry conditions: Hyperammonemic Encephalopathy. Basic parameters: up to 1 year · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Belgium
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
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.
Interventions
- Diagnostic test user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemia
user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemia, based on kinetic modelling
Primary outcome measures
- serum ammonium levels [Time frame: 1 year]
Secondary outcome measures (1)
- Removal of treatment sodium benzoate, L-arginine [Time frame: 1 year]
Eligibility criteria
Inclusion criteria
- severe acute neonatal hyperammonaemia for which acute hemodialysis is indicated
- inborn error of metabolism
- below 1 year of age
Exclusion criteria
- older than 1 year
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Belgium · 1 center
- Ghent University Hospital - Department of Paediatric Nephrology — Ghent
Identifiers
NCT: NCT05754372 · BC-10637