NCT05556096进行中（未招募）3 期
A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel, Multicenter Study to Evaluate the Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 260
- 试验地点
- 133
- 主要终点
- Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 26
研究概览
简要总结
The purpose of this study is to evaluate the safety and efficacy of ALXN1720 for the treatment of generalized MG (gMG) in adults with autoantibodies against acetylcholine receptor (AChR).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV
- •Positive serological test for autoantibodies against AChR
排除标准
- •History of thymectomy, or any other thymic surgery within 12 months prior to Screening
- •Untreated thymic malignancy, carcinoma, or thymoma
- •History of Neisseria meningitidis infection
- •Pregnancy, breastfeeding, or intention to conceive during the course of the study
研究组 & 干预措施
Placebo
Placebo Comparator
Participants will receive placebo during the 26-week RCT period, after which they will enter the OLE period of the study and receive ALXN1720.
干预措施: Placebo (Combination Product)
ALXN1720
Experimental
Participants will receive a weight-based initial (loading) dose of ALXN1720 on Day 1, followed by weight-based maintenance treatment with ALXN1720 on Day 8 and once every week (Q1W) thereafter for a total of 26 weeks. Following this randomized controlled treatment (RCT) period, all participants will receive ALXN1720 in an open-label extension (OLE) period of 105 weeks.
干预措施: ALXN1720 (Combination Product)
结局指标
主要结局
Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 26
时间窗: Baseline, Week 26
次要结局
- Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Week 26(Baseline, Week 26)
- Percentage of Responders Based on Reduction of the MG-ADL Total Score at Week 26(Baseline up to Week 26)
- Percentage of Responders based on Reduction of the QMG Total Score at Week 26(Baseline up to Week 26)
- Change From Baseline in Myasthenia Gravis Composite (MGC) Total Score at Week 26(Baseline, Week 26)
- Change from Baseline in the QMG total score at Week 4(Baseline, Week 4)
研究者
研究点 (133)
Loading locations...
相似试验
进行中（未招募）
3 期
Study of ALXN2220 Versus Placebo in Adults With ATTR-CMTransthyretin Amyloid CardiomyopathyNCT06183931Alexion Pharmaceuticals, Inc.1,181
撤回
2 期
ALXN1830 in Patients With Warm Autoimmune Hemolytic AnemiaWarm Autoimmune Hemolytic AnemiaNCT04256148Alexion Pharmaceuticals, Inc.
进行中（未招募）
3 期
Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPPHypophosphatasiaNCT06079359Alexion Pharmaceuticals, Inc.30
终止
3 期
Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson DiseaseWilson DiseaseNCT03403205Alexion Pharmaceuticals, Inc.214
已完成
3 期
A Multi-center Study to Assess the Efficacy and Safety of LX211 in Active Non-infectious Anterior UveitisUveitis, AnteriorPanuveitisNCT00404885Lux Biosciences, Inc.108
相关资讯
AstraZeneca's Gefurulimab Meets Phase III Endpoints in Myasthenia Gravis but Lags Behind Competitor Therapies- AstraZeneca's gefurulimab achieved a statistically significant 1.6-point improvement in MG-ADL scores compared to placebo in the Phase III PREVAIL study for generalized myasthenia gravis.
- The complement 5 inhibitor demonstrated inferior efficacy compared to rival therapies, including UCB's Rystiggo which showed up to 2.6-point MG-ADL improvements and AstraZeneca's own Soliris and Ultomiris.
- Gefurulimab showed favorable safety profile with mild-to-moderate adverse events and offers subcutaneous administration, potentially positioning it to compete in the $6.1 billion gMG market as treatment paradigms shift toward self-administered therapies.10 months agoInebilizumab Shows Promise in Myasthenia Gravis, Phase 3 Data Suggests- Phase 3 MINT trial data indicates inebilizumab significantly improves Myasthenia Gravis Activities of Daily Living (MG-ADL) scores compared to placebo.
- The study included patients with both anti-acetylcholine receptor (AChR) and muscle-specific tyrosine kinase (MuSK) antibody-positive generalized myasthenia gravis (gMG).
- Amgen is planning regulatory submissions for inebilizumab as a potential new treatment option for generalized myasthenia gravis (gMG).
- Ongoing trials are exploring new therapeutic approaches, including bispecific nanoantibodies and complement inhibitors, to address unmet needs in gMG treatment.last year