[pubmed] Efficacy and safety of rozanolixizumab in moderate-to-severe generalised myasthenia gravis: A phase 2 RCT
-
Auteur du sujet - Ami(e) de Diamant
- Messages : 3070
- Enregistré le : 31 mai 2020 09:57
- 3
- Zodiaque :
- Âge : 20
- Contact :
[pubmed] Efficacy and safety of rozanolixizumab in moderate-to-severe generalised myasthenia gravis: A phase 2 RCT
Neurology. 2020 Nov 20:10.1212/WNL.0000000000011108. doi: 10.1212/WNL.0000000000011108. Online ahead of print.
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: change from baseline to day 29 in Quantitative Myasthenia Gravis (QMG) score. Secondary endpoints: change from baseline to day 29 in MG-Activities of Daily Living (MG-ADL) and MG-Composite (MGC) scores; safety.
RESULTS: Forty-three patients were randomized (rozanolixizumab: 21 patients; placebo: 22 [period 1]). Least squares (LS) mean change from baseline to day 29 for rozanolixizumab vs placebo: QMG (LS mean: -1.8 vs -1.2; difference: -0.7; 95% 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: While 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.
PMID:33219142 | DOI:10.1212/WNL.0000000000011108
Source: https://pubmed.ncbi.nlm.nih.gov/3321914 ... 0&v=2.13.0
Si vous appréciez notre travail, merci de nous soutenir un petit don en cliquant ICI
Pour obtenir la traduction en français,
cliquez sur le bouton situé dans la barre des menus en haut de cette page
Pour les donateurs, si cet article vous intéresse, nous pouvons faire l’acquisition d'un tiré-à-part.
Merci d'en faire la demande sur association.amis-modo@myasthenie.com
Bonne lecture...
Pour obtenir la traduction en français,
cliquez sur le bouton situé dans la barre des menus en haut de cette page
Pour les donateurs, si cet article vous intéresse, nous pouvons faire l’acquisition d'un tiré-à-part.
Merci d'en faire la demande sur association.amis-modo@myasthenie.com
Bonne lecture...