2023-507746-83-00招募中3 期
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, 3-Arm, 3-Period Study to Assess the Efficacy and Safety of a New Formulation of Oral Cladribine Compared with Placebo in Participants with Generalized Myasthenia Gravis (MyClad)
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 70
- 试验地点
- 64
- 主要终点
- Change from Baseline in Myasthenia Gravis - Activities of Daily Living (MG-ADL) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period.
研究概览
简要总结
To assess the clinical efficacy of cladribine capsules (high and low doses) on gMG relative to placebo.
研究设计
- 分配方式
- Randomized
- 主要目的
- N/a
- 盲法
- Double (Monitor, Investigator, Analyst, Subject)
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
- 否
入选标准
- •"Diagnosis of Myasthenia Gravis with generalized muscle weakness, meeting clinical criteria for Myasthenia Gravis Foundation of America Class II to IVa classification. - In participants positive for Acetylcholine receptor antibody (anti-AChR) or muscle-specific kinase antibody (anti-MuSK) - In participants that are autoantibody seronegative and participants who are positive for anti-low-density lipoprotein receptor-related protein 4 antibodies (anti-LRP4) "
- •Has a Screening and Baseline MG-ADL score more than or equal to (>=) 6 with at least 50 percent (%) of the total score due to non-ocular symptoms. Screening and Baseline MG-ADL scores must be stable. The difference between the screening and Baseline scores should not be more than 2 and there should be no reported MG exacerbation during the screening period.
- •If treated with oral corticosteroids: should be on a stable daily dose for at least 3 months prior to and during screening. In such case, the daily dose of oral steroids should not exceed 20 milligrams(mg)/day for prednisone/prednisolone or 16 mg/day for methylprednisolone.
- •If treated with acetylcholinesterase inhibitor should be on a stable daily dose for at least 3 months prior and during screening..
- •Have a body weight >= 40 kilograms.
- •Other protocol defined inclusion criteria could apply.
排除标准
- •Immunologic disorder other than MG or any other condition requiring chronic oral, intravenous, intramuscular, or intraarticular corticosteroid therapy. Well-controlled thyroid disease, as per the Treating Investigator or the participants regular treating physician recorded in the source documents, is not exclusionary.
- •Active malignancy, or history of cancer.
- •Treatment with nonsteroidal immunosuppressants, used in gMG, such as azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus within 4 weeks prior to randomization.
- •History of generalized seizures (except for history of febrile seizures during the participant’s childhood)
- •History of recurrent infections (that is 3 or more infections per year) within the last 2 years
- •Discontinuation of treatment with any non-steroidal immunosuppressants used in gMG, such as azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus within the last 6 months prior to Screening
- •If treated with non-steroidal immunosuppressants for gMG, the dose at Screening should not exceed 50 mg/day for azathioprine, 500 mg/day for mycophenolate mofetil, 1 mg/day for tacrolimus, 50 mg/day for cyclosporine, or 7.5 mg/week for methotrexate
- •Participation in clinical study of any investigational drug within 6 months, or 5 half-lives of the investigational drug used in the previous clinical study prior to randomization, whichever is longer. However, participants with any prior exposure to cladribine may not enter the study regardless of timing of exposure
- •Treatment with eculizumab, rozanolixizumab efgartigimod, ravulizumab, or zilucoplan within 8 weeks prior to randomization
- •History of thymectomy within 6 months prior to Screening.
- •History of myasthenic crisis in the last 12 months prior to and during screening
- •Applicable for France only: Persons under court protection, persons not affiliated to a social security system, protected adults.
- •Negative for Varicella Zoster Virus antibodies at screening.
- •Other protocol defined exclusion criteria could apply.
- •Molecularly characterized or suspected congenital myasthenic syndrome, Lambert-Eaton myasthenic syndrome, inherited myopathy, muscular dystrophy, acquired myopathy or any other neurologic or systematic disease that mimics MG muscular weakness.
- •Active, clinically significant viral, bacterial, or fungal infection, including brain MRI findings consistent with signs of infection such as PML, or any major episode of infection requiring hospitalization or treatment with parenteral anti-infectives within 8 weeks prior or during Screening, or completion of oral anti-infectives within 8 weeks prior or during Screening, or a history of recurrent infections. Vaginal candidiasis, onychomycosis, and genital or oral herpes simplex virus considered by the Investigator to be sufficiently controlled would not be exclusionary.
- •Has a history of or current diagnosis of active tuberculosis (TB).
研究组 & 干预措施
Cladribine Placebo
Placebo
干预措施: Cladribine Placebo (Drug)
cladribine
Test
干预措施: cladribine (Drug)
Shingrix powder and suspension for suspension for injection Herpes zoster vaccine (recombinant, adjuvanted)
Auxiliary
干预措施: Shingrix powder and suspension for suspension for injection Herpes zoster vaccine (recombinant, adjuvanted) (Drug)
结局指标
主要结局
Change from Baseline in Myasthenia Gravis - Activities of Daily Living (MG-ADL) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period.
Change from Baseline in Myasthenia Gravis - Activities of Daily Living (MG-ADL) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period.
次要结局
- Number of Participants with Abnormal ECG parameters. [Time Frame: Up to End of Study (Week 144)]
- Change from Baseline in Quantitative Myasthenia Gravis (QMG) Scale Score at Week 24 during the Double-Blind Placebo Controlled (DBPC) Period.
- Percentage of MG-ADL Responders at Week 24 during the Double-Blind Placebo Controlled (DBPC) Period.
- Change from Baseline in Myasthenia Gravis Composite (MGC) Scale Score at Week 24 during the Double-Blind Placebo Controlled (DBPC) Period.
- Percentage of Quantitative Myasthenia Gravis (QMG) Scale Responders at Week 24 during the Double-Blind Placebo Controlled (DBPC) Period.
- Change from Baseline in the Revised Myasthenia Gravis Quality of Life – 15 Scale (MG-Qol15r) Score at Week 24 during the Double-Blind Placebo Controlled (DBPC) Period.
- Time From Initial Cladribine Full Dose Treatment to First Retreatment of Rescue Treatment up to end of Study.
- Number of Participants With Adverse Events (AEs) and Adverse Events of Special Interest (AESIs).
- Number of participants with Adverse Events (AEs) by Severity as per National Cancer Institute Common Terminology Criteria for Adverse Events, version 5.0.
- Number of Participants with Abnormal Laboratory Variables including Absolute Lymphocyte Count and Vital Signs.
- Pharmacokinetic (PK) Plasma Concentrations of Cladribine.
研究者
Global Regulatory Affairs
Scientific
Merck Healthcare KGaA
研究点 (64)
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