A Study to Test Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis

The purpose of the MycarinGstudy is to demonstrate the clinical efficacy and to assess safety and tolerability of rozanolixizumab in patients with generalized myasthenia gravis (MG).

Sex

All

Minimum age

18 Years

Inclusion Criteria:

* Study participant must be ≥18 years of age, at the time of signing the informed consent
* Study participant has documented diagnosis of generalized myasthenia gravis (gMG) at Visit 1, based on study participant's history and supported by previous evaluations
* Study participant has a confirmed positive record of autoantibodies against acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) at Screening (Visit 1).The presence of autoantibodies may be confirmed with repeat testing at Visit 1
* Study participant has Myasthenia Gravis Foundation of America (MGFA) Class II to IVa at Visit 1
* Study participant with a Myasthenia Gravis-Activities of Daily Living (MG-ADL) score of at least 3 (with ≥3 points from non-ocular symptom) AND a quantitative myasthenia gravis (QMG) score of at least 11 at Visit 1 and at Baseline (Visit 2)
* Study participant is considered for additional treatment such as intravenous immunoglobulin g (IVIg) or plasma exchange (PEX) by the Investigator

Exclusion Criteria:

* Study participant has a known history of hyperprolinemia
* Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of investigational medicinal product (IMP)
* Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI) will be excluded
* Study participant has experienced hypersensitivity reaction after exposure to other anti-neonatal Fc receptor (FcRn) drugs
* Study participant with severe (defined as Grade 3 on the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis at Visit 1 or Visit 2
* Study participant has a history of a solid organ transplant or hematopoietic stem cell/marrow transplant

• RESULTBril V, Druzdz A, Grosskreutz J, Habib AA, Mantegazza R, Sacconi S, Utsugisawa K, Vissing J, Vu T, Boehnlein M, Bozorg A, Gayfieva M, Greve B, Woltering F, Kaminski HJ; MG0003 study team. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study. Lancet Neurol. 2023 May;22(5):383-394. doi: 10.1016/S1474-4422(23)00077-7. PMID 37059507
• RESULTHabib AA, Sacconi S, Antonini G, Cortes-Vicente E, Grosskreutz J, Mahuwala ZK, Mantegazza R, Pascuzzi RM, Utsugisawa K, Vissing J, Vu T, Wiendl H, Boehnlein M, Greve B, Woltering F, Bril V. Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis: a subgroup analysis of the randomised, double-blind, placebo-controlled, adaptive phase III MycarinG study. Ther Adv Neurol Disord. 2024 Sep 12;17:17562864241273036. doi: 10.1177/17562864241273036. eCollection 2024. PMID 39297052
• RESULTKaminski HJ, Antozzi C, Habib AA, Pascuzzi RM, Sacconi S, Utsugisawa K, Vissing J, Regnault A, Hareendran A, Grimson F, Tarancon T, Bril V; MycarinG study investigators. Improvement in Patient-Reported Symptoms of Generalised Myasthenia Gravis With Rozanolixizumab in the Randomised Phase 3 MycarinG Study Using the MG Symptoms PRO. Eur J Neurol. 2025 Aug;32(8):e70231. doi: 10.1111/ene.70231. PMID 40755069
• RESULTBarnett-Tapia C, Cortes Vicente E, Pascuzzi RM, Utsugisawa K, Bloemers J, Grimson F, Tarancon T, Bril V; MycarinG study investigators. Measuring the effect of rozanolixizumab using the Myasthenia Gravis Impairment Index: analyses from the randomized phase 3 MycarinG study. J Neurol. 2025 Nov 8;272(12):752. doi: 10.1007/s00415-025-13480-8. PMID 41205003
• RESULTBril V, Druzdz A, Grosskreutz J, Habib AA, Mantegazza R, Sacconi S, Utsugisawa K, Vu T, Boehnlein M, Grimson F, Houston N, Kerbusch V, Pulido-Valdeolivas I, Tarancon T, Vissing J. Long-term use of rozanolixizumab in generalised myasthenia gravis: final pooled analysis of the phase III MycarinG study and two open-label extensions. Ther Adv Neurol Disord. 2026 Jun 29;19:17562864261458532. doi: 10.1177/17562864261458532. eCollection 2026.