Evidence map›Paper›PMID 30219580›Full record

Trial reportPharmacological research2018

Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.

Laurie S Conklin, Jesse M Damsker, Eric P Hoffman, William J Jusko, Panteleimon D Mavroudis, Benjamin D Schwartz, Laurel J Mengle-Gaw, Edward C Smith, Jean K Mah, Michela Guglieri and 22 more

3 registry-linked trialsOpen access · hybridAbstract readClinical Trial, Phase IIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Pharmacological research, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 65 papers, 4 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
65citing papers in PubMed, 4 pooled it
4.5field-weighted citation impact, top 4% 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
NCT06713135 phase4active not recruitingnot on this mapstarted 2024, after this paper: background citation

An Open-label Study to Collect Safety and Effectiveness Information on Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy Who Have Completed Prior Studies With Vamorolone

TypeinterventionalSponsorSanthera PharmaceuticalsRan2024 to 2028Enrolled80ConditionsDuchenne Muscular DystrophyArmsvamorolone 40 mg/mL oral suspension
3 · Its place in the literature

Who cites it

65 citing papers in PubMed, 4 syntheses or guidelines pooled it, 94 citations in OpenAlex.

  1. Meta-analysis of the efficacy and safety of vamorolone in Duchenne muscular dystrophy.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025
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5 more citing papers are in PubMed but not listed here.

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

32 authors at 17 institutions in 6 countries.

Laurie S ConklinReveraGen Biopharma, LLC, Rockville, MD, USA; Children's National Health System, George Washington University, Washington, DC, USA.
Jesse M DamskerReveraGen Biopharma, LLC, Rockville, MD, USA.
Eric P HoffmanReveraGen Biopharma, LLC, Rockville, MD, USA; Binghamton University- SUNY, Binghamton, NY, USA.
William J JuskoState University of New York-Buffalo, Buffalo, NY, USA.
Panteleimon D MavroudisState University of New York-Buffalo, Buffalo, NY, USA.
Benjamin D SchwartzCamden Group, LLC, St. Louis, MO, USA.
Laurel J Mengle-GawCamden Group, LLC, St. Louis, MO, USA.
Edward C SmithDuke University, Durham, NC, USA.
Jean K MahUniversity of Calgary, Alberta Children's Hospital, Calgary, Alberta, Canada.
Michela GuglieriNewcastle University, Newcastle, UK.
Yoram NevoSchneider Children's Medical Center of Israel, Tel Aviv University, Tel Aviv, Israel.
Nancy KuntzAnn & Robert H. Lurie Children's Hospital Chicago, IL, USA.
Craig M McDonaldUniversity of California Davis, Davis, CA, USA.
Mar TuliniusQueen Silvia Children's Hospital, Gothenburg, Sweden.
Monique M RyanRoyal Children's Hospital, University of Melbourne, Melbourne, Australia.
Richard WebsterThe Children's Hospital at Westmead, Sydney, Australia.
Diana CastroUniversity of Texas Southwestern, Dallas, TX, USA.
Richard S FinkelNemours Children's Hospital, Orlando, FL, USA.
Andrea L SmithTRiNDS LLC, Kensington, MD, USA.
Lauren P MorgenrothTRiNDS LLC, Kensington, MD, USA.
Adrienne ArrietaTRiNDS LLC, Kensington, MD, USA.
Maya ShimonyTRiNDS LLC, Kensington, MD, USA.
Mark JarosSummit Analytical, Denver, CO, USA.
Phil ShaleSummit Analytical, Denver, CO, USA.
John M McCallReveraGen Biopharma, LLC, Rockville, MD, USA.
Yetrib HathoutBinghamton University- SUNY, Binghamton, NY, USA.
Kanneboyina NagarajuReveraGen Biopharma, LLC, Rockville, MD, USA; Binghamton University- SUNY, Binghamton, NY, USA.
John van den AnkerReveraGen Biopharma, LLC, Rockville, MD, USA; Children's National Health System, George Washington University, Washington, DC, USA.
Leanne M WardChildren's Hospital of Eastern Ontario, Ottawa, ON, Canada.
Alexandra AhmetChildren's Hospital of Eastern Ontario, Ottawa, ON, Canada.
Michaelyn R CornishReveraGen Biopharma, LLC, Rockville, MD, USA.
Paula R ClemensUniversity of Pittsburgh and Department of Veterans Affairs Medical Center, Pittsburgh, PA, USA. Electronic address: clempr@upmc.edu.
Binghamton University · USReveraGen BioPharma (United States) · USChildren's National · USDuke University · USUniversity at Buffalo, State University of New York · USChildren's Hospital at Westmead · AUChildren's Hospital of Eastern Ontario · CADrottning Silvias barn- och ungdomssjukhus · SELurie Children's Hospital · USNemours Children's Clinic · USNewcastle University · GBRoyal Children's Hospital · AUTel Aviv University · ILThe University of Texas Southwestern Medical Center · USUniversity of Calgary · CAUniversity of California, Davis · 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
Research_ProjectU54HD090257 · NICHD · CHILDREN'S RESEARCH INSTITUTE · PI GUAY-WOODFORD, LISA MARIE · 2016 to 2020
$5.9M
Commercialization Readiness Pilot (CRP) to maximize vamorolone international labeling and salesSB1NS095423 · NINDS · REVERAGEN BIOPHARMA, INC. · PI HOFFMAN, ERIC P. · 2020 to 2021
$3.4M
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
Dissociation of efficacy from side effects of anti-inflammatory therapies in Duchenne muscular dystrophyP50HD090254 · NICHD · CHILDREN'S RESEARCH INSTITUTE · PI VAN DEN ANKER, JOHANNES NICOLAAS · 2019 to 2020
$1.6M
VBP15, an Innovative Steroid-like Intervention on DMD: VISION-DMDU34AR068616 · NIAMS · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI CLEMENS, PAULA R · 2015 to 2015
$300k
NIAMS NIH HHS U34 AR068616NICHD NIH HHS P50 HD090254NICHD NIH HHS U54 HD090254NICHD NIH HHS U54 HD090257NINDS NIH HHS R44 NS095423NINDS NIH HHS SB1 NS095423
6 · The paper itself

Abstract

We report a first-in-patient study of vamorolone, a first-in-class dissociative steroidal anti-inflammatory drug, in Duchenne muscular dystrophy. This 2-week, open-label Phase IIa multiple ascending dose study (0.25, 0.75, 2.0, and 6.0 mg/kg/day) enrolled 48 boys with Duchenne muscular dystrophy (4 to <7 years), with outcomes including clinical safety, pharmacokinetics and pharmacodynamic biomarkers. The study design included pharmacodynamic biomarkers in three contexts of use: 1. Secondary outcomes for pharmacodynamic safety (insulin resistance, adrenal suppression, bone turnover); 2. Exploratory outcomes for drug mechanism of action; 3. Exploratory outcomes for expanded pharmacodynamic safety. Vamorolone was safe and well-tolerated through the highest dose tested (6.0 mg/kg/day) and pharmacokinetics of vamorolone were similar to prednisolone. Using pharmacodynamic biomarkers, the study demonstrated improved safety of vamorolone versus glucocorticoids as shown by reduction of insulin resistance, beneficial changes in bone turnover (loss of increased bone resorption and decreased bone formation only at the highest dose level), and a reduction in adrenal suppression. Exploratory biomarkers of pharmacodynamic efficacy showed an anti-inflammatory mechanism of action and a beneficial effect on plasma membrane stability, as demonstrated by a dose-responsive decrease in serum creatine kinase activity. With an array of pre-selected biomarkers in multiple contexts of use, we demonstrate the development of the first dissociative steroid that preserves anti-inflammatory efficacy and decreases steroid-associated safety concerns. Ongoing extension studies offer the potential to bridge exploratory efficacy biomarkers to clinical outcomes.

Indexed as

Administration, OralAnti-Inflammatory AgentsBiomarkersBlood GlucoseChildChild, PreschoolHumansHydrocortisoneInsulinMaleMuscular Dystrophy, DuchennePregnadienediolsAnti-Inflammatory AgentsBiomarkersBlood GlucoseHydrocortisoneInsulinPregnadienediolsVBP15 compound

Identifiers

PMID30219580
PMCPMC6218284
OpenAlexW2892018897

What OpenQuestion holds

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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.