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Войти в исследования JAMA neurology · 2026
Efficacy and Safety of Gefurulimab in Generalized Myasthenia Gravis: The PREVAIL Phase 3 Randomized Clinical Trial.
СтатьяРандомизированное исследованиеКлиническое исследование III фазыМногоцентровое исследованиеНазвание и аннотация: Перевод в очереди
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Gwathmey Kelly G, Saccà Francesco, Howard James F, Vu Tuan, Xi Jianying, Mäurer Mathias, Ruck Tobias, Shin Ha Young, Takahashi Masanori P, Casasnovas Carlos, Smilowski Marek, Peric Stojan, Masuda Masayuki, Scholz Joachim, Shang Shulian, Yee Min, Rakhade Sanjay, Annane Djillali
Аннотация
IMPORTANCE: Gefurulimab is a novel dual-binding nanobody that blocks complement component 5 activation. Complement activation is a key pathogenic mechanism in anti-acetylcholine receptor antibody-positive (AChR-Ab+) generalized myasthenia gravis (gMG).
OBJECTIVE: To evaluate the efficacy and safety of gefurulimab in adults with AChR-Ab+ gMG.
DESIGN, SETTING, AND PARTICIPANTS: This randomized clinical trial, PREVAIL, was a phase 3, double-blind, placebo-controlled study conducted at 113 sites in 20 countries. Patients were screened and randomized from November 2022 to November 2024; the randomized controlled treatment period was 26 weeks. Participants were adults (aged ≥18 years) with AChR-Ab+ gMG, a Myasthenia Gravis Foundation of America classification II through IV, and a Myasthenia Gravis Activities of Daily Living (MG-ADL) total score of 5 or higher.
INTERVENTION: Gefurulimab or placebo via once-weekly subcutaneous self-injection.
MAIN OUTCOMES AND MEASURES: The primary end point was change from baseline in MG-ADL total score at week 26. The key secondary end point was change from baseline in Quantitative Myasthenia Gravis (QMG) total score at week 26. Safety was also assessed.
RESULTS: Of 405 patients screened, 145 were excluded; 260 met eligibility criteria and were randomized (gefurulimab, n = 131; placebo, n = 129); 249 patients completed the 26-week randomized controlled treatment period. The mean (SD) age was 52.8 (15.73) years; 157 (60.4%) patients were female and 103 (39.6%) were male. All primary and secondary end points were met with statistical significance. Improvements occurred within 1 week for MG-ADL score and 4 weeks for QMG score and were sustained through week 26. Least-squares mean change from baseline at week 26 for MG-ADL and QMG total scores for gefurulimab vs placebo were -4.2 vs -2.6 (treatment difference, -1.6; 95% CI, -2.4 to -0.8; P < .001) and -4.5 vs -2.4 (treatment difference, -2.1; 95% CI, -3.1 to -1.1; P < .001), respectively. The incidence of adverse events (AEs) was similar between groups. Most common treatment-emergent AEs with gefurulimab were injection site reactions, headache, back pain, and nasopharyngitis. No meningococcal infections were reported.
CONCLUSIONS AND RELEVANCE: Gefurulimab demonstrated both early and sustained clinical benefit in AChR-Ab+ gMG and was well tolerated, supporting its potential as a convenient, once-weekly, self-administered treatment regimen.
TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT05556096.
OBJECTIVE: To evaluate the efficacy and safety of gefurulimab in adults with AChR-Ab+ gMG.
DESIGN, SETTING, AND PARTICIPANTS: This randomized clinical trial, PREVAIL, was a phase 3, double-blind, placebo-controlled study conducted at 113 sites in 20 countries. Patients were screened and randomized from November 2022 to November 2024; the randomized controlled treatment period was 26 weeks. Participants were adults (aged ≥18 years) with AChR-Ab+ gMG, a Myasthenia Gravis Foundation of America classification II through IV, and a Myasthenia Gravis Activities of Daily Living (MG-ADL) total score of 5 or higher.
INTERVENTION: Gefurulimab or placebo via once-weekly subcutaneous self-injection.
MAIN OUTCOMES AND MEASURES: The primary end point was change from baseline in MG-ADL total score at week 26. The key secondary end point was change from baseline in Quantitative Myasthenia Gravis (QMG) total score at week 26. Safety was also assessed.
RESULTS: Of 405 patients screened, 145 were excluded; 260 met eligibility criteria and were randomized (gefurulimab, n = 131; placebo, n = 129); 249 patients completed the 26-week randomized controlled treatment period. The mean (SD) age was 52.8 (15.73) years; 157 (60.4%) patients were female and 103 (39.6%) were male. All primary and secondary end points were met with statistical significance. Improvements occurred within 1 week for MG-ADL score and 4 weeks for QMG score and were sustained through week 26. Least-squares mean change from baseline at week 26 for MG-ADL and QMG total scores for gefurulimab vs placebo were -4.2 vs -2.6 (treatment difference, -1.6; 95% CI, -2.4 to -0.8; P < .001) and -4.5 vs -2.4 (treatment difference, -2.1; 95% CI, -3.1 to -1.1; P < .001), respectively. The incidence of adverse events (AEs) was similar between groups. Most common treatment-emergent AEs with gefurulimab were injection site reactions, headache, back pain, and nasopharyngitis. No meningococcal infections were reported.
CONCLUSIONS AND RELEVANCE: Gefurulimab demonstrated both early and sustained clinical benefit in AChR-Ab+ gMG and was well tolerated, supporting its potential as a convenient, once-weekly, self-administered treatment regimen.
TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT05556096.