Evidence map›Paper›PMID 32434278›Full record

ArticleJournal of clinical pharmacology2020

Exposure-Response Analysis of Vamorolone (VBP15) in Boys With Duchenne Muscular Dystrophy.

Xiaonan Li, Laurie S Conklin, John van den Anker, Eric P Hoffman, Paula R Clemens, William J Jusko

2 registry-linked trialsOpen access · greenAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of clinical pharmacology, 2020. 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 6 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed, 1 pooled it
0.7field-weighted citation impact, top 33% of its field
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.

NCT05166109 phase2completednot on this mapstarted 2022, after this paper: background citation

A Phase II Pilot Trial of Vamorolone vs. Placebo for the Treatment of Becker Muscular Dystrophy

TypeinterventionalSponsorReveraGen BioPharma, Inc.Ran2022 to 2025Enrolled46ConditionsBecker Muscular DystrophyArmsVamorolone, Placebo
NCT05185622 phase2completednot on this mapstarted 2022, after this paper: background citation

A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

TypeinterventionalSponsorSanthera PharmaceuticalsRan2022 to 2024Enrolled54ConditionsDuchenne Muscular DystrophyArmsVamorolone
3 · Its place in the literature

Who cites it

6 citing papers in PubMed, 1 synthesis or guideline pooled it, 13 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Article
  4. Review
  5. Article
  6. 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

6 authors at 4 institutions in 1 country.

Xiaonan LiDepartment of Pharmaceutical Sciences, University at Buffalo, State University of New York, Buffalo, New York, USA.
Laurie S ConklinReveraGen BioPharma, Rockville, Maryland, USA.
John van den AnkerReveraGen BioPharma, Rockville, Maryland, USA.
Eric P HoffmanReveraGen BioPharma, Rockville, Maryland, USA.
Paula R ClemensUniversity of Pittsburgh and Department of Veterans Affairs Medical Center, Pittsburgh, Pennsylvania, USA.
William J JuskoDepartment of Pharmaceutical Sciences, University at Buffalo, State University of New York, Buffalo, New York, USA.ORCID 0000-0003-4027-0550
Children's National · USUniversity at Buffalo, State University of New York · USBinghamton University · USUniversity of Pittsburgh Medical Center · US

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
Commercialization Readiness Pilot (CRP) to maximize vamorolone international labeling and salesSB1NS095423 · NINDS · REVERAGEN BIOPHARMA, INC. · PI HOFFMAN, ERIC P. · 2020 to 2021
$3.4M
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 CONKLIN, LAURIE S · 2016 to 2018
$2.5M
Investigation of the role of UNC-45A in Paclitaxel-mediated microtubule stabilityR01GM130800 · NIGMS · UNIVERSITY OF MINNESOTA · PI BAZZARO, MARTINA · 2019 to 2020
$614k
NICHD NIH HHS U54 HD090254NIGMS NIH HHS R01 GM130800NIGMS NIH HHS R35 GM131800NINDS NIH HHS R44 NS095423NINDS NIH HHS SB1 NS095423
6 · The paper itself

Abstract

Exposure-response relationships of vamorolone, a novel dissociative steroidal anti-inflammatory drug, were investigated in clinical trials in boys with Duchenne muscular dystrophy. Variables were clinical outcome measures, Fridericia-corrected QT (QTcF) duration, and pharmacodynamic (PD) biomarkers. Exposure metrics were area under the plasma concentration time curve (AUC) and maximum plasma concentration (C

Indexed as

Administration, OralAnti-Inflammatory AgentsArea Under CurveBiomarkersChildClinical Trials as TopicDose-Response Relationship, DrugDrug Administration ScheduleHeart Conduction SystemHumansMaleMuscular Dystrophy, DuchennePregnadienediolsTreatment OutcomeAnti-Inflammatory AgentsBiomarkersPregnadienediolsVBP15 compoundchildrenDuchenne muscular dystrophy (DMD)exposure-responseglucocorticoidspharmacodynamicsvamorolone

Identifiers

PMID32434278
PMCPMC7494537
OpenAlexW3026513007

What Socratic holds

Textmetadata
LicenceTDM
Read underepoch 390

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 Socratic graph.