# An Open Label Trial of Ixazomib for Treatment Resistant Myasthenia Gravis

> **NIH NIH U54** · GEORGE WASHINGTON UNIVERSITY · 2022 · $247,178

## Abstract

Myasthenia gravis (MG) is an autoimmune disorder characterized by muscle weakness
caused by autoantibodies, mainly targeting the muscle acetylcholine receptor (AChR) and the
muscle specific kinase (MuSK). These antibodies are produced by short-lived plasmablasts and
long-lived plasma cells. To date, no MG treatment specifically targets either of these cells.
Ixazomib is a proteasome inhibitor used successfully to treat multiple myeloma, a cancer of
malignant plasma cells. We propose the IxMG trial to evaluate whether orally administered
ixazomib will be safe and have biological and clinical signals of efficacy for patients with
treatment-resistant AChR or MuSK MG, which comprise upwards of 30% of the MG population.
Patients will be treated with 3 cycles of ixazomib (weekly infusions every week for 3 weeks)
during a period of 3 months followed by 3 month follow-up. Clinical and biological monitoring will
be performed at time of each treatment and then every 4 weeks. The trial will assess the safety,
tolerability, and activity of ixazomib as a therapy for treatment-resistant patients with generalized
myasthenia gravis and detailed autoantibody analysis, lymphocyte characterization, and
antibody repertoire evaluation. A no-go decision for a Phase 2 assessment will be made for
excess adverse effects or lack of target engagement, while a decision to proceed will be made
based on safety and confirmation of biological target engagement.

## Key facts

- **NIH application ID:** 10437798
- **Project number:** 5U54NS115054-04
- **Recipient organization:** GEORGE WASHINGTON UNIVERSITY
- **Principal Investigator:** HENRY J KAMINSKI
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $247,178
- **Award type:** 5
- **Project period:** 2019-09-01 → 2024-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10437798

## Citation

> US National Institutes of Health, RePORTER application 10437798, An Open Label Trial of Ixazomib for Treatment Resistant Myasthenia Gravis (5U54NS115054-04). Retrieved via AI Analytics 2026-05-28 from https://api.ai-analytics.org/grant/nih/10437798. Licensed CC0.

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