Evidence map›Paper›PMID 41832158›Full record

ArticleNature communications2026

Modifying muscle metabolic dysregulation in inclusion body myositis with pioglitazone: a single-arm trial.

Brittany L Adler, Michael R Bene, Cissy Zhang, Conrad Say, Pratik Khare, Christopher Mecoli, Eleni Tiniakou, Julie J Paik, Tae Chung, Erika Darrah and 8 more

Registry-linked trialAbstract readClinical Trial, Phase I
PubMed Full text at PMC Publisher
In one paragraph

Article in Nature communications, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03440034 (An Open-Label Pilot Study of Pioglitazone in Sporadic Inclusion Body Myositis), which is not on this map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–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.

NCT03440034 phase1completednot on this map

An Open-Label Pilot Study of Pioglitazone in Sporadic Inclusion Body Myositis

TypeinterventionalSponsorJohns Hopkins UniversityRan2018 to 2020Enrolled19ConditionsMyositis, Inclusion Body Myositis, Muscular Diseases, Musculoskeletal DiseaseArmsPioglitazone
3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. 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

18 authors.

Brittany L Adler *Division of Rheumatology, Department of Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA. brit.adler@jhmi.edu.ORCID http://orcid.org/0000-0001-9912-2435
Michael R Bene *Division of Geriatric Medicine and Gerontology, Johns Hopkins School of Medicine, Baltimore, MD, USA.ORCID http://orcid.org/0000-0002-5417-4052
Cissy ZhangGigantest, Inc, 31 Light Street, Baltimore, MD, USA.
Conrad SayDepartment of Biology, Johns Hopkins University, Krieger School of Arts and Sciences, Baltimore, MD, USA.
Pratik KhareGigantest, Inc, 31 Light Street, Baltimore, MD, USA.
Christopher MecoliDivision of Rheumatology, Department of Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA.
Eleni TiniakouDivision of Rheumatology, Department of Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA.
Julie J PaikDivision of Rheumatology, Department of Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA.
Tae ChungDepartment of Physical Medicine and Rehabilitation, Johns Hopkins School of Medicine, Baltimore, MD, USA.
Erika DarrahDivision of Rheumatology, Department of Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA.ORCID http://orcid.org/0000-0003-1119-0279
Thomas LloydDepartment of Neurology, Johns Hopkins School of Medicine, Baltimore, MD, USA.
Ruben PagkatipunanDepartment of Physical Medicine and Rehabilitation, Johns Hopkins School of Medicine, Baltimore, MD, USA.
Megan McGowanDepartment of Physical Medicine and Rehabilitation, Johns Hopkins School of Medicine, Baltimore, MD, USA.
Albert MearsDepartment of Physical Medicine and Rehabilitation, Johns Hopkins School of Medicine, Baltimore, MD, USA.
Hilary J VernonDepartment of Genetic Medicine, Johns Hopkins School of medicine, Baltimore, MD, USA.
Lisa Christopher-StineDivision of Rheumatology, Department of Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA.ORCID http://orcid.org/0000-0002-7150-7306
Anne LeGigantest, Inc, 31 Light Street, Baltimore, MD, USA.
Jemima AlbaydaDivision of Rheumatology, Department of Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA. jalbayd1@jhmi.edu.ORCID http://orcid.org/0000-0002-8030-9504

Funding

Multidisciplinary Rheumatology Training ProgramT32AR048522 · NIAMS · JOHNS HOPKINS UNIVERSITY · PI CLIFTON O BINGHAM, LIVIA A CASCIOLA-ROSEN · 2003 to 2026
$6.4M
Translational Aging Research Training ProgramT32AG058527 · NIA · JOHNS HOPKINS UNIVERSITY · PI Peter M. Abadir, Esther Seunghee Oh · 2018 to 2026
$4.0M
RNA: Polymerase III specific CD8+ T cells: a mechanistic insight into cancer-induced autoimmunity in sclerodermaK08AR077732 · NIAMS · UNIVERSITY OF TEXAS HLTH SCI CTR HOUSTON · PI TINIAKOU, ELENI · 2021 to 2025
$794k
Skeletal Myopathy in Systemic Sclerosis: Devising a Classification Schema Predictive of OutcomesK23AR073927 · NIAMS · JOHNS HOPKINS UNIVERSITY · PI PAIK, JULIE JISUN · 2020 to 2024
$787k
Optimizing the Approach to Identify Cancer-Associated MyositisK23AR075898 · NIAMS · JOHNS HOPKINS UNIVERSITY · PI MECOLI, CHRISTOPHER · 2019 to 2023
$773k
Thermo-Scientific Q Exactive Plus Orbitrap LC-MS/MS SystemS10OD025226 · OD · JOHNS HOPKINS UNIVERSITY · PI LE, ANNE · 2018 to 2018
$562k
Post-Infectious Dysautonomia: Insights into Clinical Phenotypes and Disease PathogenesisK23AI180356 · NIAID · JOHNS HOPKINS UNIVERSITY · PI Brittany Lee Adler · 2025 to 2026
$397k
Division of Intramural Research, National Institute of Allergy and Infectious Diseases (Division of Intramural Research of the NIAID) K23AI180356NIAID NIH HHS K23 AI180356NIAMS NIH HHS K08 AR077732NIAMS NIH HHS K23 AR073927NIAMS NIH HHS K23 AR075898NIAMS NIH HHS T32 AR048522NIA NIH HHS T32 AG058527NIH HHS S10 OD025226U.S. Department of Health & Human Services | National Institutes of Health (NIH) 1S10OD025226-01U.S. Department of Health & Human Services | NIH | National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) K08AR077732U.S. Department of Health & Human Services | NIH | National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) K23AR073927U.S. Department of Health & Human Services | NIH | National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) K23AR075898U.S. Department of Health & Human Services | NIH | National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) T32AR048522U.S. Department of Health & Human Services | NIH | National Institute on Aging (U.S. National Institute on Aging) 2T32AG058527-06A1
6 · The paper itself

Abstract

This single-arm, open-label phase 1 trial evaluated the PPARγ agonist pioglitazone in patients with inclusion body myositis (IBM). After a 16-week observation (lead-in) period, participants received pioglitazone 45 mg daily for 32 weeks. The primary outcome was the change in PPARGC1A expression and related metabolic pathways in muscle after 16 weeks of treatment compared with the lead-in period. Of the 16 enrolled participants, 13 initiated pioglitazone and completed at least one on-treatment assessment; the trial was terminated early due to the COVID-19 pandemic. At baseline, muscle metabolomics revealed broad metabolic abnormalities compared with controls. Pioglitazone reversed elements of this signature, increasing PPARGC1A expression (p = 0.099) and modulating downstream pathways in muscle, including enhanced oxidative phosphorylation. Clinical outcomes were unchanged overall, but a subset with favorable metabolic responses showed slower decline in the IBM-Functional Rating Score (IBM-FRS) and Modified Timed Up and Go (m-TUG). Reported adverse effects included myalgia and heart failure exacerbation. As a phase 1 trial with a limited cohort, these findings provide preliminary evidence that pioglitazone modulates muscle metabolism and warrants further investigation in IBM. This study was supported by the Ira T. Discovery Fund and the Peter and Carmen Lucia Buck Foundation Myositis Discovery Fund. Clinical Trials Registration: NCT03440034.

Indexed as

Muscle, SkeletalMyositis, Inclusion BodyPioglitazoneAgedCOVID-19FemaleHumansMaleMetabolomicsMiddle AgedPeroxisome Proliferator-Activated Receptor Gamma Coactivator 1-alphaPPAR gammaPPAR-gamma AgonistsTreatment OutcomePeroxisome Proliferator-Activated Receptor Gamma Coactivator 1-alphaPioglitazonePPAR gammaPPAR-gamma AgonistsPPARGC1A protein, human

Identifiers

PMID41832158
PMCPMC13136323

What OpenQuestion holds

Textmetadata
LicenceCC BY
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 OpenQuestion graph.