@article{8701608,
  abstract     = {{Objective To explore the clinical efficacy and safety of subcutaneous (SC) rozanolixizumab, an anti-neonatal Fc receptor humanized monoclonal antibody, in patients with generalized myasthenia gravis (gMG). Methods In this phase 2a, randomized, double-blind, placebo-controlled, 2-period, multicenter trial (NCT03052751), patients were randomized (1:1) in period 1 (days 1-29) to 3 once-weekly (Q1W) SC infusions of rozanolixizumab 7 mg/kg or placebo. In period 2 (days 29-43), patients were re-randomized to either rozanolixizumab 7 mg/kg or 4 mg/kg (3 Q1W SC infusions), followed by an observation period (days 44-99). Primary endpoint was change from baseline to day 29 in Quantitative Myasthenia Gravis (QMG) score. Secondary endpoints were change from baseline to day 29 in MG-Activities of Daily Living (MG-ADL) and MG-Composite (MGC) scores and safety. Results Forty-three patients were randomized (rozanolixizumab 21, placebo 22 [period 1]). Least squares (LS) mean change from baseline to day 29 for rozanolixizumab vs placebo was as follows: QMG (LS mean -1.8 vs -1.2, difference -0.7, 95% upper confidence limit [UCL] 0.8; p = 0.221; not statistically significant), MG-ADL (LS mean -1.8 vs -0.4, difference -1.4, 95% UCL -0.4), and MGC (LS mean -3.1 vs -1.2, difference -1.8, 95% UCL 0.4) scores. Efficacy measures continued to improve with rozanolixizumab 7 mg/kg in period 2. The most common adverse event in period 1 was headache (rozanolixizumab 57%, placebo 14%). Conclusion Whereas change from baseline in QMG was not statistically significant, the data overall suggest rozanolixizumab may provide clinical benefit in patients with gMG and was generally well tolerated. Phase 3 evaluation is ongoing (NCT03971422). Classification of Evidence This study provides Class I evidence that for patients with gMG, rozanolixizumab is well-tolerated, but did not significantly improve QMG score.}},
  author       = {{Bril, V and Benatar, M and Andersen, H and Vissing, J and Brock, M and Greve, B and Kiessling, P and Woltering, F and Griffin, L and Van den Bergh, P and Investigators, MG0002 and De Bleecker, Jan}},
  issn         = {{0028-3878}},
  journal      = {{NEUROLOGY}},
  language     = {{eng}},
  number       = {{6}},
  pages        = {{E853--E865}},
  title        = {{Efficacy and safety of rozanolixizumab in moderate to severe generalized myasthenia gravis : a phase 2 randomized control trial}},
  url          = {{http://doi.org/10.1212/wnl.0000000000011108}},
  volume       = {{96}},
  year         = {{2021}},
}

Altmetric

View in Altmetric

Web of Science

Times cited: