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Phase 3 Completed N=200 Randomized Quadruple-blind Treatment

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

Source: ClinicalTrials.gov NCT03971422 ↗
Enrolled (actual)
200
Serious AEs
9.0%
Results posted
Aug 2023
Primary outcomePrimary: Change From Baseline to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score — -0.784; -3.370; -3.403 units on a scale — p=<0.001
◆ Published Evidence
Highly cited
266citations · ~89 / year
Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study.
The Lancet. Neurology · 2023 · High-confidence link

Summary

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).

Linked Publications (5)

  • Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study.
    The Lancet. Neurology · 2023 · 266 citations · High-confidence link
  • 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.
    Therapeutic advances in neurological disorders · 2024 · 11 citations · Open access · High-confidence link
  • Improvement in Patient-Reported Symptoms of Generalised Myasthenia Gravis With Rozanolixizumab in the Randomised Phase 3 MycarinG Study Using the MG Symptoms PRO.
    European journal of neurology · 2025 · 2 citations · Open access · High-confidence link
  • Measuring the effect of rozanolixizumab using the Myasthenia Gravis Impairment Index: analyses from the randomized phase 3 MycarinG study.
    Journal of neurology · 2025 · 0 citations · Open access · High-confidence link
  • Clinical meaningfulness and psychometric robustness of the MG Symptoms PRO scales in clinical trials in adults with myasthenia gravis.
    Frontiers in neurology · 2024 · 6 citations · Open access · Likely link

Outcome Measures

OutcomeResultp-value
PRIMARY
Change From Baseline to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score
-0.784; -3.370; -3.403 <0.001 sig
SECONDARY
Percentage of Participants Achieving Myasthenia Gravis-Activities of Daily Living (MG-ADL) Response at Day 43
28.4; 68.2; 61.2 <0.001 sig
SECONDARY
Change From Baseline to Day 43 in Myasthenia Gravis-Composite (MG-C) Total Score
-2.029; -5.930; -7.554 <0.001 sig
SECONDARY
Change From Baseline to Day 43 in Quantitative Myasthenia Gravis (QMG) Total Score
-1.915; -5.398; -6.672 <0.001 sig
SECONDARY
Change From Baseline to Day 43 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' Score
-10.588; -23.029; -25.751 <0.001 sig
SECONDARY
Change From Baseline to Day 43 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Physical Fatigue' Score
-10.637; -19.287; -25.459 0.012 sig
SECONDARY
Change From Baseline to Day 43 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Bulbar Symptoms' Score
-3.519; -14.839; -14.224 <0.001 sig
SECONDARY
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
45; 52; 57
SECONDARY
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal of Investigational Medicinal Product (IMP)
2; 2; 4

Eligibility Criteria

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
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03971422) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.

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