Thursday 16 October, 2025
Introduction: A novel IV anti-inflammatory drug is in development for the treatment of infectious disease. Clinical proof-of-pharmacology was demonstrated in…
Read morePoster: Methodology - Study Design
Bayesian bio-comparability using small sample sizes and quantification of safety risk
Thursday 16 October, 2025
Objectives: A test compound is being developed as a potential oral anti-inflammatory. The compound will be investigated for the first…
Read moreA workflow for evaluating and optimizing the designs of paediatric studies
Thursday 16 October, 2025
Objectives: To develop a standardized in-silico workflow for efficiently designing paediatric PK studies, aligning with the FDA precision criteria for sample…
Read moreProbability of Success with Exposure Response Modelling and Clinical Trial Simulation as a Tool to Support Decision Making
Thursday 16 October, 2025
Objectives: The aims of this work are: 1) to describe the method of evaluating probability of success by applying exposure…
Read moreDose confirmation based on population pharmacokinetics within LUMINESCE (NCT04963270); a Phase III, randomized, double-blind, placebo-controlled study evaluating satralizumab in patients with generalized myasthenia gravis
Thursday 16 October, 2025
Objectives: Satralizumab is a humanized IgG2 monoclonal recycling IL-6 receptor antagonist antibody. It has been approved for treatment of neuromyelitis…
Read moreSparse sampling design for characterizing individual PK of recombinant factor VIII fusion protein (rFVIIIFc) in prophylactic treatment of Hemophilia A
Thursday 16 October, 2025
Objectives: To determine sparse sampling times for estimating individual rFVIIIFc maximum a priori (MAP) Bayesian estimates in children (<12yr) and…
Read moreA clinical trial simulation study to investigate and compare efficacy and toxicity findings in parallel and titration designs
Thursday 16 October, 2025
Background & Objectives: Dose-ranging studies are designed to understand the efficacy and toxicity of an investigational compound, aiming to maximise…
Read moreModel-based adaptive optimal design for nonlinear mixed-effects models with model structure uncertainty. Application in a simulated dose-finding study.
Thursday 16 October, 2025
Objectives: Model-based adaptive optimal design (MBAOD) is a promising methodological tool for designing efficient dose-finding studies. MBAODs are allowed to…
Read moreUse of PBPK information to select the doses and criteria for early dose confirmation or adjustment with a minimum number of subjects in a pediatric proof of concept (POC) study
Thursday 16 October, 2025
Objectives: DrugA is a small molecule being developed for a neurologic disorder in a pediatric target population. Dose selection for…
Read moreAssessing the adequacy of a minimal PK sampling schedule for individual dose adjustment decisions in a proof of mechanism (PoM) study in a special population
Thursday 16 October, 2025
Objectives: Drug A was previously tested in healthy subjects and patients and is now being investigated in a proof of…
Read more