John Mondick

Bioequivalence of ruxolitinib once-daily extended-release vs twice-daily immediate-release tablets in healthy adults

Xiaohua Gong, Zhiyin Xun, Jay Getsy, Ryan McGee et autres

Abstract Background: Ruxolitinib is an oral Janus kinase (JAK)1/JAK2 inhibitor approved for the treatment of adults with intermediate/high-risk myelofibrosis or polycythemia vera who are resistant or intolerant to hydroxyurea, as well as patients aged ≥12 years with steroid-refractory acute graft-versus-host disease (GVHD) …

us (code pays fourni par la source)

Bayesian Population Pharmacokinetic Modeling of Ondansetron for Neonatal Opioid Withdrawal Syndrome

Kevin Lam, John Mondick, Gary Peltz, Manhong Wu et autres

Ondansetron is an anti-emetic 5-HT3 receptor antagonist being investigated for treating neonatal opioid withdrawal syndrome (NOWS). Sparse PK data were analyzed from a multicenter, double-blind clinical trial with 98 mother/neonate dyads. Pregnant women with opioid use disorder were randomized to receive either …

us (code pays fourni par la source)

Corrigendum: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease

Barry J. Byrne, Giancarlo Parenti, Benedikt Schoser, Ans T. van der Ploeg et autres

Corrigendum on: Byrne BJ, Parenti G, Schoser B, van der Ploeg AT, Do H, Fox B, Goldman M, Johnson FK, Kang J, Mehta N, Mondick J, Sheikh MO, Sitaraman Das S, Tuske S, Brudvig J, Weimer JM, Mozaffar T. Cipaglucosidase alfa plus …

us, it, de, nl (code pays fourni par la source)

Pharmacokinetics and Pharmacodynamics of an Antibody Targeting Pathological Tau for the Treatment of Alzheimer’s Disease: Nonclinical Studies in P301S Mice and Cynomolgus Macaques

Chanchal Sadhu, Wencheng Liu, J. K. Kundu, Jeffery S. Thompson et autres

Abstract Background VY‐TAU01 is a recombinant humanized IgG4 monoclonal antibody (mAb) directed against pathological tau for the treatment of patients with mild dementia or mild cognitive impairment due to Alzheimer’s disease (AD). Both VY‐TAU01 and its parental mouse IgG1 mAb Ab‐01 target …

us (code pays fourni par la source)

Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease

Barry J. Byrne, Giancarlo Parenti, Benedikt Schoser, Ans T. van der Ploeg et autres

Enzyme replacement therapy (ERT) is the only approved disease-modifying treatment modality for Pompe disease, a rare, inherited metabolic disorder caused by a deficiency in the acid α-glucosidase (GAA) enzyme that catabolizes lysosomal glycogen. First-generation recombinant human GAA (rhGAA) ERT (alglucosidase alfa) can …

us, it, de, nl (code pays fourni par la source)

Population pharmacokinetics of total and unbound valemetostat and platelet dynamics in healthy volunteers and patients with non‐Hodgkin lymphoma

Masato Fukae, Kyle Baron, Masaya Tachibana, John Mondick et autres

Valemetostat is an EZH2/1 inhibitor that has been approved in Japan for the treatment of patients with relapsed/refractory adult T-cell leukemia/lymphoma, based mainly on results from a single-arm phase II trial. It is currently under investigation worldwide for the treatment of other …

jp, us (code pays fourni par la source)

Use of Model-informed Drug Development and Natural History Data to Inform the Development of Iluzanebart in ALSP: A Neurological Rare Disease

François Gaudreault, Jack Cook, Donald G. McLaren, John Mondick et autres

Bayesian estimation in NONMEM

Curtis Johnston, Timothy Waterhouse, Matthew Wiens, John Mondick et autres

Bayesian estimation is a powerful but underutilized tool for answering drug development questions. In this tutorial, the principles of Bayesian model development, assessment, and prior selection will be outlined. An example pharmacokinetic (PK) model will be used to demonstrate the implementation of …

us (code pays fourni par la source)

Population Pharmacokinetic-Pharmacodynamic Modeling of Variable Wear Times for a Dextroamphetamine Transdermal System

Mariacristina Castelli, Marina Komaroff, Suzanne Meeves, Kanan Balakrishnan et autres

Abstract Introduction The Dextroamphetamine Transdermal System (d-ATS) was developed as an alternative to oral amphetamine (AMP) formulations for ADHD. In a pivotal study, d-ATS met primary and secondary efficacy endpoints for ADHD in children and adolescents. Study subjects wore d-ATS for 9 …

us (code pays fourni par la source)

3.1 Population Pharmacokinetic-Pharmacodynamic Modeling of Variable Wear Times for a Dextroamphetamine Transdermal System

Suzanne Meeves, Mariacristina Castelli, Marina Komaroff, Kanan Balakrishnan et autres

PB2247: PHARMACOKINETIC MODELING AND SIMULATION TO SUPPORT MITAPIVAT DOSE SELECTION USED IN PEDIATRIC PHASE III STUDIES

Ophelia Yin, Kyle Baron, John Mondick, Meredith Little et autres

Background: Mitapivat (AG-348) is a first-in-class, oral, small molecule, allosteric activator of the red blood cell pyruvate kinase (PK) enzyme (PKR). The positive benefit-risk profile of mitapivat has been demonstrated in two recently completed phase 3 studies in adults with PK deficiency …

us (code pays fourni par la source)

Bayesian modeling and simulation to inform rare disease drug development early decision-making: Application to Duchenne muscular dystrophy

Janelle L. Lennie, John Mondick, Marc R. Gastonguay

Rare disease clinical trials are constrained to small sample sizes and may lack placebo-control, leading to challenges in drug development. This paper proposes a Bayesian model-based framework for early go/no-go decision making in rare disease drug development, using Duchenne muscular dystrophy (DMD) …

us (code pays fourni par la source)