2023-510007-22-00招募中2 期
An Open-label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Inebilizumab in Pediatric Subjects with Neuromyelitis Optica Spectrum Disorder
Horizon Therapeutics Ireland Designated Activity Company5 个研究点 分布在 5 个国家目标入组 5 人开始时间: 2024年5月14日最近更新:
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 5
- 试验地点
- 5
- 主要终点
- 1. PK parameters, including maximum observed concentration (Cmax), area under the concentration-time curve from time 0 to 14 days post dose (AUC0-14d) and from time 0 extrapolated to infinity (AUC0-inf), systemic clearance, terminal elimination half-life (t½), and volume of distribution at steady state (Vss)
研究概览
简要总结
- To characterize the PK of inebilizumab administered in pediatric subjects with NMOSD
- To characterize the PD of inebilizumab administered in pediatric subjects with NMOSD
- To assess the safety and tolerability of inebilizumab administered in pediatric subjects with NMOSD
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Follow-up period
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Written informed consent and any locally required data privacy authorization obtained from the subject's legally authorized representative in accordance with regional laws or regulations and the subject's assent, when applicable, prior to performing any protocolrelated procedures.
- •Male or female subjects, minimum body weight of 15 kg, age 2 to < 18 years at the time of screening.
- •Positive serum anti-AQP4-IgG result at screening (verified by the XML File Identifier: /BYZ1q6d7Yxs391VjDKysjqZIlE=Page 11/27 central laboratory) and diagnosed with NMOSD
- •Documented history of one or more NMOSD acute relapses within the last year, or 2 or more NMOSD acute relapses within 2 years prior to screening.
- •Female subjects of childbearing potential who are sexually active with a nonsterilized male partner must agree to use a highly effective method of contraception from screening until 6 months after the final dose of investigational product (IP)
- •Nonsterilized male subjects who are sexually active with a female partner of childbearing potential must agree to use a male condom from Day 1 until 3 months after the final dose of IP.
排除标准
- •Any condition that, in the opinion of the Investigator, would interfere with the evaluation or administration of the IP or interpretation of subject safety or study results
- •Concurrent/previous enrollment in another clinical study involving an investigational treatment within 4 weeks or 5 published half-lives of the investigational treatment, whichever is the longer, prior to Day 1
- •Females who are breastfeeding, pregnant, or who intend to become pregnant at any time from screening until 6 months after the final dose of IP
- •Known history of allergy or reaction to any component of the IP formulation or history of anaphylaxis following any biologic therapy
- •Evidence of alcohol, drug, or chemical abuse, or a recent history of such abuse < 1 year prior to Day 1
- •Major surgery within 8 weeks prior to screening
- •Spontaneous or induced abortion, still or live birth, or pregnancy ≤ 4weeks prior to screening
- •Evidence of significant hepatic, renal, or metabolic dysfunction or significant hematological abnormality
- •Receipt of rituximab or any experimental B-cell depleting agent within 6 months prior to screening unless B-cell counts have returned to ≥ onehalf the LLN
- •Receipt of intravenous immunoglobulin (IVIG) within one month prior to Day 1
- •Receipt of particular immunosuppressive therapy within 2 months prior to Day 1
- •Receipt of natalizumab (Tysabri®) within 6 months prior to Day 1
- •Severe drug allergic history or anaphylaxis to 2 or more food products or medicine
- •Diagnosed with a concurrent autoimmune disease that is uncontrolled (unless approved by the medical monitor)
- •Receipt of any of the following: a. Any live or attenuated vaccine within 4 weeks prior to Day 1 b. Bacillus Calmette-Guérin vaccine within one year of screening c. Blood transfusion within 4 weeks prior to screening or during screening
- •Clinically significant serious active or chronic viral, bacterial, or fungal infection that requires treatment with anti-infectives, within 2 months prior to Day 1
- •Known history of congenital or acquired immunodeficiency that predisposes the subject to infection
- •Positive test for chronic hepatitis B infection at screening
- •Positive test for hepatitis C virus antibody
- •Negative test for varicella zoster virus (VZV)-IgG
- •History of cancer, apart from squamous cell or basal cell carcinoma of the skin treated with documented success of curative therapy > 3 months prior to Day 1
- •History of active or latent tuberculosis
- •For subjects who may undergo MRI scans: Unable to undergo an MRI scan (eg, hypersensitivity to Gd containing MRI contrast agents,implanted pacemakers, defibrillators, or other metallic objects on or inside the body that limit performing MRI scans), or unable to tolerate or comply with the MRI procedure.
结局指标
主要结局
1. PK parameters, including maximum observed concentration (Cmax), area under the concentration-time curve from time 0 to 14 days post dose (AUC0-14d) and from time 0 extrapolated to infinity (AUC0-inf), systemic clearance, terminal elimination half-life (t½), and volume of distribution at steady state (Vss)
1. PK parameters, including maximum observed concentration (Cmax), area under the concentration-time curve from time 0 to 14 days post dose (AUC0-14d) and from time 0 extrapolated to infinity (AUC0-inf), systemic clearance, terminal elimination half-life (t½), and volume of distribution at steady state (Vss)
2. CD20-positive B-cell counts on Days 1, 8, 15, 29, 57, 85, 113, 155, and 197
2. CD20-positive B-cell counts on Days 1, 8, 15, 29, 57, 85, 113, 155, and 197
3. Safety and tolerability assessments, including incidence of adverse events (AEs)/serious adverse events (SAEs)/adverse events of special interest (AESIs) and changes in laboratory parameters and vital signs
3. Safety and tolerability assessments, including incidence of adverse events (AEs)/serious adverse events (SAEs)/adverse events of special interest (AESIs) and changes in laboratory parameters and vital signs
次要结局
- 4. Change in EDSS
- 5. Presence of anti-drug antibody (ADA)
- 1. Disease activity endpoints include: − Time to first relapse − Proportion of relapse-free subjects − Annualized relapse rate
- 2. HRQoL endpoints include: − Change in Euro Quality of Life-5 Dimension Youth (EQ-5D-Y) score − Change in Pediatric Quality of Life Inventory (PedsQL)
- 3. Change in visual acuity
研究者
Nishi Rampal
Scientific
Horizon Therapeutics Ireland Designated Activity Company
研究点 (5)
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