Optimalisation of the Treatment of Acute Neonatal Hyperammonaemia

Last updated: April 11, 2024
Sponsor: University Hospital, Ghent
Overall Status: Active - Recruiting

Phase

N/A

Condition

Hepatic Encephalopathy

Urea Cycle Disorder

Hyperargininemia

Treatment

user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemia

Clinical Study ID

NCT05754372
BC-10637
  • Ages < 1
  • All Genders

Study 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. 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • severe acute neonatal hyperammonaemia for which acute hemodialysis is indicated
  • inborn error of metabolism
  • below 1 year of age

Exclusion

Exclusion Criteria:

  • older than 1 year

Study Design

Total Participants: 10
Treatment Group(s): 1
Primary Treatment: user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemia
Phase:
Study Start date:
January 15, 2023
Estimated Completion Date:
December 31, 2026

Connect with a study center

  • Ghent University Hospital - Department of Paediatric Nephrology

    Ghent, 9000
    Belgium

    Active - Recruiting

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