Evidence map›Paper›PMID 25381300›Full record

Trial reportNeurology2014

Treatment of sporadic inclusion body myositis with bimagrumab.

Anthony A Amato, Kumaraswamy Sivakumar, Namita Goyal, William S David, Mohammad Salajegheh, Jens Praestgaard, Estelle Lach-Trifilieff, Anne-Ulrike Trendelenburg, Didier Laurent, David J Glass and 3 more

Registry-linked trialOpen access · bronzeAbstract readRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Neurology, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01423110 (A Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of BYM338 in Patients With Sporadic Inclusion Body Myositis), which is not on this map. Cited by 104 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
104citing papers in PubMed, 3 pooled it
9.3field-weighted citation impact, top 1% 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.

NCT01423110 phase2completednot on this map

A Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of BYM338 in Patients With Sporadic Inclusion Body Myositis

TypeinterventionalSponsorNovartis PharmaceuticalsRan2011 to 2012Enrolled14ConditionsSporadic Inclusion Body MyositisArmsBYM338, Placebo
3 · Its place in the literature

Who cites it

104 citing papers in PubMed, 3 syntheses or guidelines pooled it, 198 citations in OpenAlex.

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  11. 15-PGDH inhibition promotes muscle repair and strength recovery during GLP-1 receptor agonist-induced weight loss.Proceedings of the National Academy of Sciences of the United States of America · 2026
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44 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

13 authors at 5 institutions in 1 country.

Anthony A AmatoFrom Brigham and Women's Hospital and Harvard Medical School (A.A.A., M.S., S.A.G.), Boston; Boston Children's Hospital (S.A.G.); Harvard-Massachusetts Institute of Technology (S.A.G.), Division of Health Sciences and Technology, Cambridge, MA; Barrow Neurological Institute (K.S.), Phoenix AZ; Massachusetts General Hospital (N.G., W.S.D.), Boston; Novartis Institutes for Biomedical Research (E.L.-T., A.-U.T., D.L., D.J.G., R.R., B.S.T.), Cambridge, MA and Basel, Switzerland; and Novartis Pharmaceuticals Corporation (J.P.), East Hanover, NJ.
Kumaraswamy SivakumarFrom Brigham and Women's Hospital and Harvard Medical School (A.A.A., M.S., S.A.G.), Boston; Boston Children's Hospital (S.A.G.); Harvard-Massachusetts Institute of Technology (S.A.G.), Division of Health Sciences and Technology, Cambridge, MA; Barrow Neurological Institute (K.S.), Phoenix AZ; Massachusetts General Hospital (N.G., W.S.D.), Boston; Novartis Institutes for Biomedical Research (E.L.-T., A.-U.T., D.L., D.J.G., R.R., B.S.T.), Cambridge, MA and Basel, Switzerland; and Novartis Pharmaceuticals Corporation (J.P.), East Hanover, NJ.
Namita GoyalFrom Brigham and Women's Hospital and Harvard Medical School (A.A.A., M.S., S.A.G.), Boston; Boston Children's Hospital (S.A.G.); Harvard-Massachusetts Institute of Technology (S.A.G.), Division of Health Sciences and Technology, Cambridge, MA; Barrow Neurological Institute (K.S.), Phoenix AZ; Massachusetts General Hospital (N.G., W.S.D.), Boston; Novartis Institutes for Biomedical Research (E.L.-T., A.-U.T., D.L., D.J.G., R.R., B.S.T.), Cambridge, MA and Basel, Switzerland; and Novartis Pharmaceuticals Corporation (J.P.), East Hanover, NJ.
William S DavidFrom Brigham and Women's Hospital and Harvard Medical School (A.A.A., M.S., S.A.G.), Boston; Boston Children's Hospital (S.A.G.); Harvard-Massachusetts Institute of Technology (S.A.G.), Division of Health Sciences and Technology, Cambridge, MA; Barrow Neurological Institute (K.S.), Phoenix AZ; Massachusetts General Hospital (N.G., W.S.D.), Boston; Novartis Institutes for Biomedical Research (E.L.-T., A.-U.T., D.L., D.J.G., R.R., B.S.T.), Cambridge, MA and Basel, Switzerland; and Novartis Pharmaceuticals Corporation (J.P.), East Hanover, NJ.
Mohammad SalajeghehFrom Brigham and Women's Hospital and Harvard Medical School (A.A.A., M.S., S.A.G.), Boston; Boston Children's Hospital (S.A.G.); Harvard-Massachusetts Institute of Technology (S.A.G.), Division of Health Sciences and Technology, Cambridge, MA; Barrow Neurological Institute (K.S.), Phoenix AZ; Massachusetts General Hospital (N.G., W.S.D.), Boston; Novartis Institutes for Biomedical Research (E.L.-T., A.-U.T., D.L., D.J.G., R.R., B.S.T.), Cambridge, MA and Basel, Switzerland; and Novartis Pharmaceuticals Corporation (J.P.), East Hanover, NJ.
Jens PraestgaardFrom Brigham and Women's Hospital and Harvard Medical School (A.A.A., M.S., S.A.G.), Boston; Boston Children's Hospital (S.A.G.); Harvard-Massachusetts Institute of Technology (S.A.G.), Division of Health Sciences and Technology, Cambridge, MA; Barrow Neurological Institute (K.S.), Phoenix AZ; Massachusetts General Hospital (N.G., W.S.D.), Boston; Novartis Institutes for Biomedical Research (E.L.-T., A.-U.T., D.L., D.J.G., R.R., B.S.T.), Cambridge, MA and Basel, Switzerland; and Novartis Pharmaceuticals Corporation (J.P.), East Hanover, NJ.
Estelle Lach-TrifilieffFrom Brigham and Women's Hospital and Harvard Medical School (A.A.A., M.S., S.A.G.), Boston; Boston Children's Hospital (S.A.G.); Harvard-Massachusetts Institute of Technology (S.A.G.), Division of Health Sciences and Technology, Cambridge, MA; Barrow Neurological Institute (K.S.), Phoenix AZ; Massachusetts General Hospital (N.G., W.S.D.), Boston; Novartis Institutes for Biomedical Research (E.L.-T., A.-U.T., D.L., D.J.G., R.R., B.S.T.), Cambridge, MA and Basel, Switzerland; and Novartis Pharmaceuticals Corporation (J.P.), East Hanover, NJ.
Anne-Ulrike TrendelenburgFrom Brigham and Women's Hospital and Harvard Medical School (A.A.A., M.S., S.A.G.), Boston; Boston Children's Hospital (S.A.G.); Harvard-Massachusetts Institute of Technology (S.A.G.), Division of Health Sciences and Technology, Cambridge, MA; Barrow Neurological Institute (K.S.), Phoenix AZ; Massachusetts General Hospital (N.G., W.S.D.), Boston; Novartis Institutes for Biomedical Research (E.L.-T., A.-U.T., D.L., D.J.G., R.R., B.S.T.), Cambridge, MA and Basel, Switzerland; and Novartis Pharmaceuticals Corporation (J.P.), East Hanover, NJ.
Didier LaurentFrom Brigham and Women's Hospital and Harvard Medical School (A.A.A., M.S., S.A.G.), Boston; Boston Children's Hospital (S.A.G.); Harvard-Massachusetts Institute of Technology (S.A.G.), Division of Health Sciences and Technology, Cambridge, MA; Barrow Neurological Institute (K.S.), Phoenix AZ; Massachusetts General Hospital (N.G., W.S.D.), Boston; Novartis Institutes for Biomedical Research (E.L.-T., A.-U.T., D.L., D.J.G., R.R., B.S.T.), Cambridge, MA and Basel, Switzerland; and Novartis Pharmaceuticals Corporation (J.P.), East Hanover, NJ.
David J GlassFrom Brigham and Women's Hospital and Harvard Medical School (A.A.A., M.S., S.A.G.), Boston; Boston Children's Hospital (S.A.G.); Harvard-Massachusetts Institute of Technology (S.A.G.), Division of Health Sciences and Technology, Cambridge, MA; Barrow Neurological Institute (K.S.), Phoenix AZ; Massachusetts General Hospital (N.G., W.S.D.), Boston; Novartis Institutes for Biomedical Research (E.L.-T., A.-U.T., D.L., D.J.G., R.R., B.S.T.), Cambridge, MA and Basel, Switzerland; and Novartis Pharmaceuticals Corporation (J.P.), East Hanover, NJ.
Ronenn RoubenoffFrom Brigham and Women's Hospital and Harvard Medical School (A.A.A., M.S., S.A.G.), Boston; Boston Children's Hospital (S.A.G.); Harvard-Massachusetts Institute of Technology (S.A.G.), Division of Health Sciences and Technology, Cambridge, MA; Barrow Neurological Institute (K.S.), Phoenix AZ; Massachusetts General Hospital (N.G., W.S.D.), Boston; Novartis Institutes for Biomedical Research (E.L.-T., A.-U.T., D.L., D.J.G., R.R., B.S.T.), Cambridge, MA and Basel, Switzerland; and Novartis Pharmaceuticals Corporation (J.P.), East Hanover, NJ.
Brian S TsengFrom Brigham and Women's Hospital and Harvard Medical School (A.A.A., M.S., S.A.G.), Boston; Boston Children's Hospital (S.A.G.); Harvard-Massachusetts Institute of Technology (S.A.G.), Division of Health Sciences and Technology, Cambridge, MA; Barrow Neurological Institute (K.S.), Phoenix AZ; Massachusetts General Hospital (N.G., W.S.D.), Boston; Novartis Institutes for Biomedical Research (E.L.-T., A.-U.T., D.L., D.J.G., R.R., B.S.T.), Cambridge, MA and Basel, Switzerland; and Novartis Pharmaceuticals Corporation (J.P.), East Hanover, NJ. sagreenberg@partners.org brian.tseng@novartis.com.
Steven A GreenbergFrom Brigham and Women's Hospital and Harvard Medical School (A.A.A., M.S., S.A.G.), Boston; Boston Children's Hospital (S.A.G.); Harvard-Massachusetts Institute of Technology (S.A.G.), Division of Health Sciences and Technology, Cambridge, MA; Barrow Neurological Institute (K.S.), Phoenix AZ; Massachusetts General Hospital (N.G., W.S.D.), Boston; Novartis Institutes for Biomedical Research (E.L.-T., A.-U.T., D.L., D.J.G., R.R., B.S.T.), Cambridge, MA and Basel, Switzerland; and Novartis Pharmaceuticals Corporation (J.P.), East Hanover, NJ. sagreenberg@partners.org brian.tseng@novartis.com.
Barrow Neurological Institute · USHarvard University · USBoston Children's Hospital · USBrigham and Women's Hospital · USMassachusetts General Hospital · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveTo study activin signaling and its blockade in sporadic inclusion body myositis (sIBM) through translational studies and a randomized controlled trial.

methodsWe measured transforming growth factor β signaling by SMAD2/3 phosphorylation in muscle biopsies of 50 patients with neuromuscular disease (17 with sIBM). We tested inhibition of activin receptors IIA and IIB (ActRII) in 14 patients with sIBM using one dose of bimagrumab (n = 11) or placebo (n = 3). The primary outcome was the change in right thigh muscle volume by MRI at 8 weeks. Lean body mass, strength, and function were secondary outcomes. Twelve of the patients (10 bimagrumab, 2 placebo) participated in a subsequent 16-week observation phase.

resultsMuscle SMAD2/3 phosphorylation was higher in sIBM than in other muscle diseases studied (p = 0.003). Eight weeks after dosing, the bimagrumab-treated patients increased thigh muscle volume (right leg +6.5% compared with placebo, p = 0.024; left leg +7.6%, p = 0.009) and lean body mass (+5.7% compared with placebo, p = 0.014). Subsequently, bimagrumab-treated patients had improved 6-minute walking distance, which peaked at 16 weeks (+14.6%, p = 0.008) compared with placebo. There were no serious adverse events; the main adverse events with bimagrumab were mild acne and transient involuntary muscle contractions.

conclusionsTransforming growth factor β superfamily signaling, at least through ActRII, is implicated in the pathophysiology of sIBM. Inhibition of ActRII increased muscle mass and function in this pilot trial, offering a potential novel treatment of sIBM. CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that for patients with inclusion body myositis, bimagrumab increases thigh muscle volume at 8 weeks.

Indexed as

Activin Receptors, Type IIAdultAgedAged, 80 and overAntibodies, BlockingAntibodies, MonoclonalAntibodies, Monoclonal, HumanizedDouble-Blind MethodExercise TestFemaleHumansImmunologic FactorsMagnetic Resonance ImagingMaleMiddle AgedMuscle, SkeletalActivin Receptors, Type IIAntibodies, BlockingAntibodies, MonoclonalAntibodies, Monoclonal, HumanizedbimagrumabImmunologic FactorsSmad2 ProteinSMAD2 protein, humanSmad3 ProteinSMAD3 protein, human

Identifiers

PMID25381300
PMCPMC4277670
OpenAlexW2163067250

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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.