NCT07142343招募中4 期
An Open-Label, Single-Arm Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Pozelimab in Pediatric Patients 1 to 5 Years of Age With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Incidence of Treatment-Emergent Adverse Event (TEAEs)
研究概览
简要总结
This study is researching a drug called pozelimab (called "study drug"). The main aim of this study is to monitor the safety and tolerability of the study drug.
The study is focused on young children 1 to 5 years of age, who have CHAPLE disease. CHAPLE is a very rare hereditary disease that can cause potentially life-threatening symptoms related to the stomach and intestines (gastrointestinal symptoms), and symptoms related to the heart and blood vessels (cardiovascular symptoms).
The study is also looking at several other research questions, including:
- What side effects may happen from taking the study drug
- How much study drug is in the blood at different times
- Whether the study drug blocks Complement 5 (C5) in the body
- Whether the study drug changes the level of a substance called CH50 measured in the blood
- Whether the study drug changes the levels of albumin and other proteins
- Whether the body makes antibodies against study drug, which could make the study drug less effective or could lead to side effects
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of CD55-deficient CHAPLE disease as described in the protocol
- •Parent(s)/legal guardian(s) are willing and able to comply with participant's clinic visits and study-related procedures, including participant's completion of the full series of meningococcal vaccinations required per protocol.
- •Parent(s)/legal guardian(s) are willing to provide written informed consent
- •Participant has active CHAPLE disease as described in the protocol
排除标准
- •History of meningococcal infection
- •No documented meningococcal quadrivalent (serotype ACWY) vaccination prior to screening and participant's parent(s)/legal guardian(s) are unwilling for participant to undergo vaccination during the study as described in the protocol
- •No documented vaccination for Haemophilus influenzae and Streptococcus pneumoniae if applicable based on local practice or guidelines prior to screening as described in the protocol
- •Prior treatment with a complement inhibitor as described in the protocol
- •Presence of a concomitant disease that leads to hypoproteinemia or secondary intestinal lymphangiectasia as described in the protocol
- •Note: Other Protocol Defined Inclusion/ Exclusion Criteria Apply
研究组 & 干预措施
Active CHAPLE
Experimental
干预措施: Pozelimab (Drug)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Event (TEAEs)
时间窗: Through week 52
Severity of TEAEs
时间窗: Through week 52
次要结局
- Concentrations of total pozelimab(Through week 52)
- Concentrations of total C5 in serum(Through week 52)
- Absolute change from baseline of albumin concentration in serum(Through week 52)
- Percent change from baseline of albumin concentration in serum(Through week 52)
- Absolute change from baseline of total protein(Through week 52)
- Percent change from baseline of total protein(Through week 52)
- Absolute change from baseline of total Immunoglobulin G (IgG)(Through week 52)
- Percent change from baseline of total IgG(Through week 52)
- Incidence of Anti-Drug Antibody (ADA) to pozelimab over time(Through week 52)
- Titer of ADA to pozelimab over time(Through week 52)
- Change from baseline of total complement activity CH50 over time(Through week 52)
- Percent change from baseline of CH50 over time(Through week 52)
研究者
研究点 (1)
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