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NCT Number: NCT05754372

Optimalisation of the Treatment of Acute Neonatal Hyperammonaemia

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.

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Key information

Age range

Up to 1 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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

Diagnostic Test

user-friendly and widely applicable algorithm to tailor the treatment of acute neonatal hyperammonemia, based on kinetic modelling

Primary outcomes

  1. serum ammonium levels

    Time frame: 1 year

    serum ammonium levels

Secondary outcomes

  1. Removal of treatment sodium benzoate, L-arginine

    Time frame: 1 year

    serum and dialysate concentration (mg/dL) of sodium benzoate and L-arginine

Study contacts

Contact information is provided by the study sponsor or research team.

Evelien Snauwaert, MD, PhD

CONTACT

[email protected]

00 32 9 332 69 05

Sunny Eloot, PhD

CONTACT

[email protected]

00 32 9 332 10 25

Sponsors and collaborators

Lead sponsor

University Hospital, Ghent

Other

Registry information

Important dates

Study start
2023
Primary completion
2025
Study completion
2026
First posted
Mar 3, 2023
Registry last updated
Apr 15, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.