2023-508842-17-00招募中3 期
Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Monotherapy in Patients with Symptomatic Generalized Myasthenia Gravis
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 118
- 试验地点
- 30
- 主要终点
- Change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score
研究概览
简要总结
To evaluate the effect of pozelimab + cemdisiran and cemdisiran monotherapy on daily functioning that is impacted by signs and symptoms in patients with symptomatic generalized myasthenia gravis (gMG)
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Male or female patients ≥18 years of age at screening (or ≥ legal age of adulthood based on local regulations, whichever is older)
- •Patient with documented diagnosis of myasthenia gravis (MG) based on medical history and supported by previous evaluations as described in the protocol
- •Documented prior history of positive serologic test or a positive result during screening of anti-acetylcholine receptor (AChR) antibodies or anti-LRP4 antibodies
- •Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class II to IVa at screening
- •Myasthenia Gravis-Activities of Daily Living (MG-ADL) score ≥6 at screening. Ocular items should not contribute more than 50% of MG-ADL total score as described in the protocol.
- •Currently receiving an acetylcholinesterase inhibitor or documented reason for not using acetylcholinesterase inhibitor therapy per investigator
- •Currently receiving an immunosuppressive therapy (IST) for MG, or documented reason why the patient is not taking an IST per investigator
- •If currently receiving an IST, not anticipated to have IST dosage changed before randomization or during double-blind treatment period (DBTP)
- •Willing and able to comply with clinic visits and study-related procedures, including completion of the primary series of the meningococcal vaccinations required per protocol
排除标准
- •Patients with antibody profile that is only positive for MuSK (MuSK positivity is based on a documented prior history of positive serologic test for antibodies to MuSK or a positive result during screening)
- •History of thymectomy within 12 months prior to screening or planned during the study
- •History of malignant thymoma (patients with stage 1 may be enrolled), or history of cancer within the past 5 years, except for adequately treated basal cell skin cancer, squamous cell skin cancer, or in situ cervical cancer
- •Myasthenic crisis or Myasthenia Gravis Foundation of America (MGFA) Class V within 1 month of screening
- •Not meeting meningococcal vaccination requirements and, at a minimum, documentation of quadrivalent meningococcal vaccination within 5 years prior to randomization and serotype B vaccine (when available) within 3 years prior to randomization as described in the protocol.
- •Known contraindication to meningococcal vaccines (group ACWY conjugate and group B vaccines) as described in the protocol
- •Patients who require antibiotics for meningococcal prophylaxis and have a contraindication, warning, or precaution precluding the use of penicillin class and penicillin-alternative antibiotics planned to be used for prophylaxis, or a history of intolerance leading to the discontinuation of these antibiotics
- •Positive hepatitis B surface antigen or hepatitis C virus ribonucleic acid (RNA) during screening
- •History of HIV infection or a positive test at screening per local requirements NOTE: Other protocol-defined Inclusion/ Exclusion Criteria apply
结局指标
主要结局
Change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score
Change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score
次要结局
- Change from baseline in Quantitative Myasthenia Gravis (QMG) score
- Achievement of a ≥3-point reduction (improvement) in MG-ADL total score
- Achievement of a ≥5-point reduction (improvement) in QMG total score
- Achievement of a consistent response on the MG-ADL
- Achievement of minimal symptom expression (MSE)
- Change from baseline in the Myasthenia Gravis Composite (MGC) total score
- Change from baseline in Myasthenia Gravis Quality of Life (MG QOL15r) total score
- Achievement of a ≥2-, 4-, 5-, 6-, 7-, 8-, 9-, or 10-point reduction on MG-ADL total score
- Achievement of a ≥3-, 4-, 6-, 7-, 8-, 9-, or 10-point reduction on QMG
- Incidence and severity of treatment-related adverse events (TEAEs) in patients treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo
- Incidence and severity of serious adverse events (SAEs) in patients treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo
- Incidence and severity of adverse events of special interest (AESIs) in patients treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo
- Concentrations of total pozelimab in serum
- Concentrations of total complement component 5 (C5) in plasma
- Concentrations of cemdisiran and its metabolites in plasma
- Incidence of treatment-emergent anti-drug antibodies (ADAs) to pozelimab over time
- Incidence of treatment-emergent ADAs to cemdisiran over time
- Change in total complement hemolysis activity assay (CH50) over time
- Percent change in CH50 over time
研究者
Clinical Trial Information
Scientific
Regeneron Pharmaceuticals Inc.
研究点 (30)
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