Evidence map›Paper›PMID 38682893›Full record

ArticleJournal of clinical pharmacology2024

Assessing Pharmacogenomic loci Associated with the Pharmacokinetics of Vamorolone in Boys with Duchenne Muscular Dystrophy.

Xiaonan Li, Daniele Sabbatini, Elena Pegoraro, Luca Bello, Paula Clemens, Michela Guglieri, John van den Anker, Jesse Damsker, John McCall, Utkarsh J Dang and 2 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of clinical pharmacology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

12 authors.

Xiaonan LiDepartment of Pharmaceutical Sciences, University at Buffalo, State University of New York, Buffalo, NY, USA.ORCID 0000-0003-4039-4940
Daniele SabbatiniDepartment of Neurosciences, University of Padova, Padua, Italy.
Elena PegoraroDepartment of Neurosciences, University of Padova, Padua, Italy.
Luca BelloDepartment of Neurosciences, University of Padova, Padua, Italy.
Paula ClemensDepartment of Neurology, University of Pittsburgh, Pittsburgh, PA, USA.
Michela GuglieriJohn Walton Centre for Neuromuscular Disease, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle-upon-Tyne, UK.
John van den AnkerDivision of Clinical Pharmacology, Children's National Hospital, Washington, DC, USA.
Jesse DamskerReveraGen BioPharma, Rockville, MD, USA.
John McCallReveraGen BioPharma, Rockville, MD, USA.
Utkarsh J DangDepartment of Health Sciences, Carleton University, Ottawa, Canada.
Eric P HoffmanReveraGen BioPharma, Rockville, MD, USA.
William J JuskoDepartment of Pharmaceutical Sciences, University at Buffalo, State University of New York, Buffalo, NY, USA.ORCID 0000-0003-4027-0550

Funding

Pivotal trial of vamorolone in Duchenne muscular dystrophyR44NS095423 · NINDS · REVERAGEN BIOPHARMA, INC. · PI CLEMENS, PAULA R, HOFFMAN, ERIC P. · 2016 to 2019
$6.1M
Mechanistic Pharmacokinetics and PharmacodynamicsR35GM131800 · NIGMS · STATE UNIVERSITY OF NEW YORK AT BUFFALO · PI JUSKO, WILLIAM J. · 2019 to 2023
$2.9M
Dissociation of efficacy from side effects of anti-inflammatory therapies in Duchenne muscular dystrophyU54HD090254 · NICHD · CHILDREN'S RESEARCH INSTITUTE · PI VAN DEN ANKER, JOHANNES NICOLAAS · 2016 to 2018
$2.5M
VBP15, an Innovative Steroid-like Intervention on DMD: VISION-DMDU34AR068616 · NIAMS · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI CLEMENS, PAULA R · 2015 to 2015
$300k
National Institute of Child Health and Human Development 5U54HD090254National Institutes of Health, the National Institute of Neurological Disorders and Stroke R44NS095423NIAMS NIH HHS U34 AR068616NIAMS NIH HHS U34AR068616NICHD NIH HHS U54 HD090254NIGMS NIH HHS R35 GM131800NIH HHS R35-GM131800NINDS NIH HHS R44 NS095423US Department of Defense, CDMRP W81XWH-22-1-0668
6 · The paper itself

Abstract

Human genetic variation (polymorphisms) in genes coding proteins involved in the absorption, distribution, metabolism, and elimination (ADME) of drugs can have a strong effect on drug exposure and downstream efficacy and safety outcomes. Vamorolone, a dissociative steroidal anti-inflammatory drug for treating Duchenne muscular dystrophy (DMD), primarily undergoes oxidation by CYP3A4 and CYP3A5 and glucuronidation by UDP-glucuronosyltransferases. This work assesses the pharmacokinetics (PKs) of vamorolone and sources of interindividual variability (IIV) in 81 steroid-naïve boys with DMD aged 4 to <7 years old considering the genetic polymorphisms of CYPS3A4 (CYP3A4*22, CYP3A4*1B), CYP3A5 (CYP3A5*3), and UGT1A1 (UGT1A1*60) utilizing population PK modeling. A one-compartment model with zero-order absorption (T

Indexed as

Cytochrome P-450 CYP3AGlucuronosyltransferaseMuscular Dystrophy, DuchenneChildChild, PreschoolHumansMaleModels, BiologicalPharmacogeneticsPolymorphism, GeneticPregnadienediolsUGT1A1 EnzymeCYP3A4 protein, humanCYP3A5 protein, humanCytochrome P-450 CYP3AGlucuronosyltransferasePregnadienediolsUGT1A1 EnzymeVBP15 compoundDuchenne muscular dystrophypharmacogenomicspopulation pharmacokinetic modelingvamorolone

Identifiers

PMID38682893
PMCPMC11357888

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

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.