Trial reportJournal of clinical pharmacology2016
Sampling Optimization in Pharmacokinetic Bridging Studies: Example of the Use of Deferiprone in Children With β-Thalassemia.
Trial report in Journal of clinical pharmacology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 10 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Dose Rationale for Dapagliflozin and Empagliflozin in Paediatric Heart Failure: a Phase II.a Pharmacokinetics, Ease-of-swallow, Safety and Proof-of-concept Study Among Children 6-18 Years of Age
Repurposing Empagliflozin for Duchenne Muscular Dystrophy - Associated Cardiomyopathy: a Pharmacokinetics, Safety and Proof-of-concept Study Among Children 6-18 Years of Age
Who cites it
10 citing papers in PubMed, 12 citations in OpenAlex.
- Dose rationale and pharmacokinetics of dexmedetomidine in mechanically ventilated new-borns: impact of design optimisation.European journal of clinical pharmacology · 2019Trial
- Population pharmacokinetics and dosing recommendations for the use of deferiprone in children younger than 6 years.British journal of clinical pharmacology · 2017Trial
- Repurposing Empagliflozin for Duchenne Muscular Dystrophy-Associated Cardiomyopathy: Protocol for a Pharmacokinetics, Safety and Proof-of-Concept Trial in Children.Cardiology and therapy · 2026Article
- Design Optimization for Developing Population Pharmacokinetic Models in Critically Ill Children: Application to Teicoplanin, Piperacillin and Meropenem.Clinical pharmacokinetics · 2026Article
- Empagliflozin in paediatric heart failure: model-based optimisation of a pharmacokinetic bridging study.Frontiers in medicine · 2025Article
- Optimizing the design of a pharmacokinetic trial to evaluate the dosing scheme of a novel tuberculosis drug in children living with or without HIV.CPT: pharmacometrics & systems pharmacology · 2024Article
- The Vital Role Played by Deferiprone in the Transition of Thalassaemia from a Fatal to a Chronic Disease and Challenges in Its Repurposing for Use in Non-Iron-Loaded Diseases.Pharmaceuticals (Basel, Switzerland) · 2023Review
- Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia.The Cochrane database of systematic reviews · 2023Review
- Article
- How to optimise drug study design: pharmacokinetics and pharmacodynamics studies introduced to paediatricians.The Journal of pharmacy and pharmacology · 2017Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 1 institution in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Despite wide clinical experience with deferiprone, the optimum dosage in children younger than 6 years remains to be established. This analysis aimed to optimize the design of a prospective clinical study for the evaluation of deferiprone pharmacokinetics in children. A 1-compartment model with first-order oral absorption was used for the purposes of the analysis. Different sampling schemes were evaluated under the assumption of a constrained population size. A sampling scheme with 5 samples per subject was found to be sufficient to ensure accurate characterization of the pharmacokinetics of deferiprone. Whereas the accuracy of parameters estimates was high, precision was slightly reduced because of the small sample size (CV% >30% for Vd/F and KA). Mean AUC ± SD was found to be 33.4 ± 19.2 and 35.6 ± 20.2 mg · h/mL, and mean Cmax ± SD was found to be 10.2 ± 6.1 and 10.9 ± 6.7 mg/L based on sparse and frequent sampling, respectively. The results showed that typical frequent sampling schemes and sample sizes do not warrant accurate model and parameter identifiability. Expectation of the determinant (ED) optimality and simulation-based optimization concepts can be used to support pharmacokinetic bridging studies. Of importance is the accurate estimation of the magnitude of the covariate effects, as they partly determine the dose recommendation for the population of interest.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.