Evidence map›Paper›PMID 41180124›Full record

ArticleTherapeutic advances in neurological disorders2025

Open-label study of efgartigimod in seronegative myasthenia gravis.

Mohamed Khateb, Ajith Sivadasan, Carolina Barnett-Tapia, Lubna Daniyal, Lahiru Fernando, Shiyi Chen, Leif Erik Lovblom, Hans Katzberg, Vera Bril

Registry-linked trialAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Therapeutic advances in neurological disorders, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06587867 (Efficacy, Safety and Tolerability of Efgartigimod in Patients with Seronegative Generalized Myasthenia Gravis), which is not on this map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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.

NCT06587867 phase3recruitingnot on this map

Efficacy, Safety and Tolerability of Efgartigimod in Patients with Seronegative Generalized Myasthenia Gravis: an Open-Label Study

TypeinterventionalSponsorUniversity Health Network, TorontoRan2023 to 2025Enrolled30ConditionsEfgartigimod, Generalized Myasthenia GravisArmsefgartigimod
3 · Its place in the literature

Who cites it

5 citing papers in PubMed.

  1. Trial
  2. Review
  3. Article
  4. Article
  5. 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

9 authors.

Mohamed KhatebEllen and Martin Prosserman Centre for Neuromuscular Diseases, University Health Network, University of Toronto, Toronto, ON, Canada.ORCID https://orcid.org/0000-0002-2262-2674
Ajith SivadasanEllen and Martin Prosserman Centre for Neuromuscular Diseases, University Health Network, University of Toronto, Toronto, ON, Canada.
Carolina Barnett-TapiaEllen and Martin Prosserman Centre for Neuromuscular Diseases, University Health Network, University of Toronto, Toronto, ON, Canada.
Lubna DaniyalEllen and Martin Prosserman Centre for Neuromuscular Diseases, University Health Network, University of Toronto, Toronto, ON, Canada.
Lahiru FernandoEllen and Martin Prosserman Centre for Neuromuscular Diseases, University Health Network, University of Toronto, Toronto, ON, Canada.
Shiyi ChenBiostatistics Department, University Health Network, Toronto, ON, Canada.
Leif Erik LovblomInstitute of Health Policy, Management and Evaluation, University of Toronto, Toronto, ON, Canada.
Hans KatzbergEllen and Martin Prosserman Centre for Neuromuscular Diseases, University Health Network, University of Toronto, Toronto, ON, Canada.
Vera BrilEllen and Martin Prosserman Centre for Neuromuscular Diseases, University Health Network, 5EC-309, Toronto General Hospital, University of Toronto, 200 Elizabeth Street, Toronto, ON M5G 2C4, Canada.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: The ADAPT trial demonstrated the benefit of efgartigimod, a neonatal Fc receptor (FcRn) inhibitor, in acetylcholine receptor antibody (AChRAb) positive patients with generalized myasthenia gravis (MG). Information regarding the benefits in those lacking pathogenic antibodies is sparse. Objectives: We aimed to investigate the safety and efficacy of efgartigimod in patients with double-seronegative (SN) generalized MG. Design: An open-label 6-month prospective study, conducted at our center. Methods: Patients aged at least 18 years with clinical and electrodiagnostic features of MG and negative results for AChRAb and muscle-specific tyrosine kinase antibodies were included. Efgartigimod was administered weekly for 4 weeks and then biweekly for 5 months followed by an observation period. The primary endpoint was the change in MG impairment index (MGII) at 6 months compared to baseline. Secondary endpoints include the change in MG activities of daily living (MG-ADL), other MG scores, overall responders, and early responders. The safety analysis included all patients who received at least one dose of efgartigimod. Results: We enrolled 30 patients with SN MG who were resistant to other treatments and had unacceptable MGII scores. The MGII decreased by 11.92 points ( Conclusion: Efgartigimod was well tolerated and efficacious in patients with SN MG. Future randomized, placebo-controlled studies are needed. Trial registration: This trial is registered at ClinicalTrials.gov (NCT06587867), accessed via https://clinicaltrials.gov/study/NCT06587867?locStr=Toronto,%20ON,%20Canada&country=Canada&state=Ontario&city=Toronto&cond=Myasthenia%20Gravis&intr=efgartigimod&rank=1.

Indexed as

efgartigimodmyasthenia gravisseronegativestudytreatment

Identifiers

PMID41180124
PMCPMC12579174

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.