2022-501925-19-00招募中3 期
A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Abcuro Inc.
- 入组人数
- 24
- 试验地点
- 4
- 主要终点
- For Part B: The primary endpoint of the study will be mean change from Baseline (Day 1) in IBMFRS at W76. For Part C: The endpoint for Part C of the study will be PD recovery as assessed by the time from EOT/ETV to recovery of KLRG1+ cells.
研究概览
简要总结
Part A (Sentinel Cohort): NA for EU
Part B (Double-Blind Safety and Efficacy Cohort): Primary Efficacy Objectives To determine the efficacy of ABC008 in IBM at two SC dose levels as measured by IBM Functional Rating Scale (IBMFRS) at Week (W)76. Safety Objectives To determine the safety and tolerability of SC ABC008 in subjects with IBM.
Part C (PD Recovery Cohort): To evaluate the PD recovery of the depletion of killer cell lectin-like receptor G1 (KLRG1)+ cells after the end of treatment with SC ABC008 in subjects with IBM.
研究设计
- 分配方式
- Randomized
- 主要目的
- Treatment Period (Part B)
- 盲法
- Double (Analyst, Investigator, Monitor, 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
- 是
入选标准
- •Adult males and females age >40 years at the time of the first dose of study medication;
- •Able to read, understand, and provide signed informed consent prior to the performance of any study-related procedures;
- •Diagnosis of either clinico-pathologically defined IBM, clinically defined IBM, or probable IBM according to the ENMC IBM 2011 research diagnostic criteria Rose et al., 2013, modified to allow inclusion of subjects with age of onset ≥40 years documented IBM. Documented histopathology results must be available prior to Baseline (Day 1) to confirm eligibility.
- •Weight >40 and <150 kg;
- •Diagnosis of either clinico-pathologically defined IBM, clinically defined IBM, or probable IBM according to the ENMC IBM 2011 research diagnostic criteria modified to allow inclusion of subjects with age of onset ≥40 years (Rose et al., 2013). Documented histopathology results must be available prior to Baseline (Day 1) to confirm eligibility;
- •As required to complete the mTUG test, able to arise from a tandard armchair (described in Section 6.2.6, with use of their arms but without support from another person or device (e.g., cane, walking stick), at Screening and Baseline (Day 1);
- •As required to complete the mTUG test, able to walk three meters, turn around, walk back to the chair, and sit down, with or without an assistive device. Once arisen from the chair, subject may use any walking device but cannot be supported by another person, furniture, or a wall.;
- •Agree to adhere to relevant local guidelines regarding minimizing exposure to severe acute respiratory syndrome coronavirus 2 from the first Screening Visit until EOT/ETV;
- •Negative coronavirus disease 2019 (COVID-19) test results within 48 hours before Baseline (Day 1) (polymerase chain reaction [PCR] or rapid antigen testing as per local guidance);
- •Agree (to the best of their knowledge), to avoid contact with any person suspected or known to have an infectious disease of which they are at risk of contracting from the first Screening Visit until the EOT/ETV;
- •Women of childbearing potential (WOCBP) and male subjects with female partners who are WOCBP (based on gender assignation at birth) must agree to use highly effective (<1% failure rate) contraception (as detailed in protocol section 7.6.4) for at least 30 days prior to the first dose of study medication, during the study, and for 180 days following EOT/ETV;
- •Male subjects (based on gender assignation at birth) must refrain from sperm donation for the duration of the study and for 180 days after EOT/ETV;
- •WOCBP (based on gender assignation at birth) must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline (Day 1).
排除标准
- •Any other form of myositis or myopathy other than IBM, e.g., metabolic or drug-induced myopathy, drug-induced myositis, anti-synthetase syndrome, polymyositis or dermatomyositis, cancer-associated myositis (myositis diagnosed within three years, either before or after), myositis in overlap with another autoimmune disease (e.g., systemic lupus, systemic sclerosis, rheumatoid arthritis), or muscular dystrophy;
- •Any condition, e.g., severe degenerative arthritis with limited range of motion, which precludes the ability to quantitate muscle strength or perform functional assessments (e.g., mTUG), in the Investigator’s opinion;
- •Presence of another autoimmune or autoinflammatory disease which, in the view of the Principal Investigator or MM, may impact the subject’s ability to perform study procedures such that it would confound clinical assessment. Examples include rheumatoid arthritis, psoriatic arthritis, axial spondyloarthropathy, inflammatory bowel disease, systemic lupus erythematosus. Subjects with Sjogren’s syndrome, T-cell large granular lymphocyte leukemia (T-LGLL), or well-controlled thyroid disease are permitted
- •Receipt of any of the following treatments within the following time frames before Screening (or as otherwise specified): a. Intravenous or intramuscular corticosteroids: four weeks; b. Oral prednisone (or prednisone equivalent) >7.5 mg/day at Screening; c. Use of nonbiologic immunosuppressants (e.g., cyclosporine, mycophenolate mofetil or sodium, azathioprine, methotrexate, sirolimus, Janus kinase inhibitors): 12 weeks; d. Intra-articular therapies, such as corticosteroids or hyaluronic acid preparations: four weeks; e. Intravenous, intramuscular, or SC immunoglobulin (IVIG, IMIG, or SCIG): 12 weeks; f. Cytokine, integrin, or activin antagonists or selective co-stimulation modulators, including but not limited to, interleukin (IL)-1, IL-6, IL-17, IL-12/23, IL-23, interferon, integrin, and tumor necrosis factor α antagonists, abatacept, bimagrumab: 12 weeks; g. Cell-depleting or modulating therapies (e.g., alemtuzumab, antithymocyte globulin, atacicept, belimumab, obinutuzumab, ocrelizumab, ofatumumab, rituximab, siplizumab): 12 months; h. Use of chemotherapeutic regimens or other cytotoxic therapies, e.g., cyclophosphamide, doxorubicin or hydroxyrubicin, vincristine, and prednisone; purine analogs, alkylating agents: 24 weeks; i. Other immunomodulatory biologics: 12 weeks or five half-lives, whichever is longer; j. Transfusion with blood, packed red blood cells, or platelets or treatment with plasmapheresis or plasma exchange: six weeks; k. Anabolic steroids: four weeks; l. Growth hormone: four weeks; m. Physiologic supplementation with testosterone permitted provided dose stable for four weeks before Screening and expected to stay stable during the study; n. Therapies associated with neuromuscular side effects including antimalarial drugs (e.g., chloroquine, hydroxychloroquine), colchicine, cholesterol-lowering drugs (e.g., statins) except if on stable dose for at least 12 weeks, and without impact on assessment of IBM progression;
- •Initiation of an exercise or a physical therapy regimen within four weeks of Screening. Any exercise or physical therapy regimen initiated prior to the Screening Visit must have been stable for at least four weeks prior to Screening; Please refer to protocol for a complete list of the exclusion criteria
结局指标
主要结局
For Part B: The primary endpoint of the study will be mean change from Baseline (Day 1) in IBMFRS at W76. For Part C: The endpoint for Part C of the study will be PD recovery as assessed by the time from EOT/ETV to recovery of KLRG1+ cells.
For Part B: The primary endpoint of the study will be mean change from Baseline (Day 1) in IBMFRS at W76. For Part C: The endpoint for Part C of the study will be PD recovery as assessed by the time from EOT/ETV to recovery of KLRG1+ cells.
次要结局
- Efficacy: 1. Mean change from Baseline (Day 1) in MMT 12 at W76; 2. Mean change from Baseline (Day 1) in hand grip strength by dynamometry at W76; Safety: 1. Incidence, nature, and severity of TEAEs; 2. Incidence, nature, and severity of TESAEs; Please refer to protocol for a complete list of the efficacy and safety secondary Endpoints.
研究者
Garry Weems
Scientific
Abcuro Inc.
研究点 (4)
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