CTRI/2025/04/085012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
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 264
- 试验地点
- 8
- 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
The purpose of this clinical study is to determine the efficacy and safety of a new oral cladribine formulation in participants with Generalized Myasthenia Gravis (gMG) in comparison to placebo. It will also investigate the sustained efficacy, the need for retreatment, and the long-term safety of oral cladribine in gMG. An additional component is included to characterize the Pharmacokinetics (PK) of the new cladribine formulation in gMG participants. This study is divided into 3 periods: the double-blind placebo control (DBPC) pivotal period, and 2 extensions, the blinded extension (BE) and the retreatment (RT) period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Other
- 盲法
- Participant, Investigator, Outcome Assessor and Date-entry Operator Blinded
入排标准
- 年龄范围
- 18.00 Year(s) 至 99.00 Year(s)（—）
- 性别
- All
入选标准
- •Adults of more than or equal to 18 years of age at the time of signing the informed consent.
- •Diagnosis of Myasthenia Gravis with generalized muscle weakness, meeting clinical criteria for MGFA 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 mg per day for prednisone or prednisolone or 16 mg per day for methylprednisolone.
- •If treated with acetylcholinesterase inhibitor should be on a stable daily dose (pyridostigmine dose less than or equal to 480 mg per day) for at least 3 months prior to and during screening.
- •Have a body weight more than or equal to 40 kg.
- •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
- •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.
- •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)
- •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
- •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 generalized seizures (except for history of infantile febrile seizures)
- •Negative for Varicella Zoster Virus antibodies at screening History of myasthenic crisis in the last 12 months prior to and during screening
- •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
- •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
- •Other protocol defined exclusion criteria could apply.
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
时间窗: Baseline, Week 24
次要结局
- 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(At Week 24)
- Change from Baseline in Myasthenia Gravis Composite (MGC) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period(Baseline, Week 24)
- Percentage of Quantitative Myasthenia Gravis (QMG) Scale Responders at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period(At Week 24)
- Time From Initial Cladribine Full Dose Treatment to First Retreatment of Rescue Treatment up to end of Study(Up to End of Study (Week 144))
- Number of Participants With Adverse Events (AEs) and Adverse Events of Special Interest (AESIs)(Up to End of Study (Week 144))
- Number of participants with Adverse Events (AEs) by Severity as per National Cancer Institute Common Terminology Criteria for Adverse Events, version 5.0(Up to End of Study (Week 144))
- Number of Participants with Abnormal Laboratory Variables including Absolute Lymphocyte Count and Vital Signs(Up to End of Study (Week 144))
- Pharmacokinetic (PK) Plasma Concentration of Cladribine(Pre-dose, 0.25, 0.5, 1, 2, 3, 4, 6, 8 and 24 hours post-dose)
- 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(Baseline, Week 24)
研究者
研究点 (8)
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