NCT04209634已完成2 期
An Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Percentage of Participants With Active Disease at Baseline Who Achieved Normalization of Serum Albumin and Improvement in Prespecified Clinical Outcomes at Week 24
研究概览
简要总结
The primary objective of the study is to determine the effect of pozelimab on active CD55-deficient protein-losing enteropathy (PLE; CHAPLE).
The secondary objectives of the study are:
- To evaluate the safety and tolerability of pozelimab in patients with CD55-deficient PLE disease
- To evaluate the effect of pozelimab on CD55-deficient PLE (both patients with active disease at baseline and those with inactive disease on eculizumab, switching to pozelimab)
- To determine the effects of pozelimab on albumin and other serum proteins (total protein, immunoglobulins)
- To determine the effects of pozelimab on ascites
- To determine the effects of pozelimab on stool consistency
- To determine the effect of pozelimab on health-related quality of life
- To determine the effect of pozelimab on lab abnormalities observed in CD55-deficient PLE such as hypertriglyceridemia, thrombocytosis, and hypovitaminosis B12
- To describe the effects of pozelimab on the sparing of concomitant medications and reduction in hospitalization days
- To determine the effects of pozelimab on growth
- To characterize the concentration of pozelimab in patients with CD55-deficient PLE
- To assess the incidence of treatment-emergent ADA for pozelimab in patients with CD55-deficient PLE disease
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 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of CD55-deficient PLE/CHAPLE disease (based on a history of PLE), confirmed by biallelic CD55 loss-of-function mutation detected by genotype analysis
- •Active disease as defined by the protocol or inactive disease on eculizumab therapy (and whose treating physician has the expectation of future access to renewed eculizumab treatment should this be required), and is willing to discontinue eculizumab during screening and start pozelimab at baseline with no eculizumab wash-out
排除标准
- •History of meningococcal infection
- •No documented meningococcal vaccination within 3 years prior to screening and patient unwilling to undergo vaccination during the study
- •No documented vaccination for Haemophilus influenzae and Streptococcus pneumoniae if applicable based on local practice or guidelines prior to screening and patient unwilling to undergo vaccination during the study if required per local practice or guidelines
- •Presence of a concomitant disease that leads to hypoproteinemia at the time of starting pozelimab
- •A concomitant disease that leads to secondary intestinal lymphangiectasia such as a fontan procedure for congenital heart disease
- •Note: Other protocol-defined Inclusion/Exclusion criteria apply.
研究组 & 干预措施
Active PLE
Experimental
Patients aged 1 year and older with a clinical diagnosis of CD55-deficient PLE disease
干预措施: Pozelimab (Drug)
结局指标
主要结局
Percentage of Participants With Active Disease at Baseline Who Achieved Normalization of Serum Albumin and Improvement in Prespecified Clinical Outcomes at Week 24
时间窗: At Week 24
Normalization of serum albumin was defined as serum albumin within the normal range at least 70 percent (%) of measurements between weeks 12 and 24, and no single albumin measurement of \<2.5 grams per deciliter (g/dL) between weeks 12 and 24, and no requirement for albumin infusion between weeks 12 and 24. Improvement in the following 4 prespecified clinical outcomes that were evaluable for improvement at baseline, without worsening of the others: Daily bowel movement frequency, the presence and severity of facial edema (physician-reported), the presence and severity of peripheral edema (physician-reported), and the participant/caregiver assessment of frequency of problematic abdominal pain. Percentage of participants with active disease at baseline who achieved normalization of serum albumin and improvement in prespecified clinical outcomes at Week 24 were reported.
次要结局
- Percentage of Participants With Active Disease at Baseline Who Maintained Disease Control(Weeks 12 to 48; Weeks 12 to 144; Weeks 24 to 48; Weeks 48 to 96; Weeks 96 to 144)
- Change From Baseline in Physician Assessment of Facial Edema Based on a 5-point Likert Rating Scale(Baseline and Week 144)
- Change From Baseline in Physician Assessment of Peripheral Edema Based on a 5-point Likert Rating Scale(Baseline and Week 144)
- Change From Baseline in Food and Drink Limitations as Assessed by the PedsQL™ GI Symptom Scales' Food and Drink Limits Sub-scale(Baseline and Week 144)
- Change From Baseline in Albumin Values(Baseline and Week 144)
- Percentage Change From Baseline in Albumin Values(Baseline and Week 144)
- Time to First Normalization for Albumin Values(Baseline up to Week 144)
- Change From Baseline in Protein Values(Baseline and Week 144)
- Time to First Normalization for Total Protein(Baseline up to Week 144)
- Change From Baseline in Health-Related Quality of Life (HRQoL) as Assessed by the PedsQL™ Generic Core Scales(Baseline and Week 144)
- Number of Participants With Albumin Infusion by 24 Week Periods(Weeks 0 to 24; Weeks 24 to 48; Weeks 48 to 72; Weeks 72 to 96; Weeks 96 to 120; Weeks 120 to 144)
- Change From Baseline in IgG Values(Baseline and Week 144)
- Change From Baseline in Immunoglobulin (Ig) Values(Baseline and Week 144)
- Time to First Normalization for Ig Values(Baseline up to Week 144)
- Time to First Normalization for IgG Values(Baseline up to Week 144)
- Change From Baseline in IgM Values(Baseline and Week 144)
- Time to First Normalization for IgM Values(Baseline up to Week 44)
- Change From Baseline in IgA Values(Baseline and Week 144)
- Time to First Normalization for IgA Values(Baseline up to Week 144)
- Change From Baseline in Vitamin B12 Values(Baseline and Week 144)
- Time to First Normalization for Vitamin B12 Values(Baseline up to Week 144)
- Change From Baseline in Vitamin B9 (Folate) Values(Baseline and Week 144)
- Time to First Normalization for Vitamin B9 (Folate) Values(Baseline up to Week 144)
- Change From Baseline in Iron Values(Baseline and Week 144)
- Time to First Normalization for Iron Values(Baseline up to Week 144)
- Change From Baseline in Unsaturated Iron Binding Capacity(Baseline and Week 144)
- Time to First Normalization for Unsaturated Iron Binding Capacity(Baseline up to Week 144)
- Change From Baseline in Ferritin Values(Baseline and Week 144)
- Time to First Normalization for Ferritin Values(Baseline up to Week 144)
- Change From Baseline in Magnesium Values(Baseline and Week 144)
- Time to First Normalization for Magnesium Values(Baseline up to Week 144)
- Change From Baseline in Fasting Cholesterol Values(Baseline and Week 144)
- Time to First Normalization for Fasting Cholesterol Values(Baseline up to Week 144)
- Change From Baseline in Fasting Triglycerides Values(Baseline and Week 144)
- Time to First Normalization for Fasting Triglycerides Values(Baseline up to Week 144)
- Number of Participants Who Used Concomitant Medication(Baseline up to Week 144)
- Number of Hospitalization Days by 24 Week Period(Weeks 0 to 24; Weeks 24 to 48; Weeks 48 to 72; Weeks 72 to 96; Weeks 96 to 120; Weeks 120 to 144)
- Change From Baseline in Body Weight Z-Score(Baseline and Week 144)
- Change From Baseline in Height Z-Score(Baseline and Week 144)
- Change From Baseline in Total Complement Activity Complement Hemolytic Assay (CH50)(Baseline and Week 144)
- Concentrations of Total Pozelimab in Serum(Baseline up to Week 164)
- Number of Participants With Treatment-emergent Anti-drug Antibodies (ADA) to Pozelimab(Baseline up to Week 164)
- Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Severity of TEAEs(From start of study drug administration up to approximately 144 weeks)
- Number of Participants With Improvement in Most Bothersome Signs and Symptoms at Week 24(At Week 24)
- Absolute Values of Protein, and Immunoglobulin G (IgG) at Baseline and Week 24(Baseline, Week 24)
- Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24(Baseline, Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11,12,13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24)
- Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24(Baseline, Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11,12,13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24)
- Number of Participants With Abdominal Ascites at Week 24(Baseline up to Week 24)
- Absolute Value of Albumin at Specified Timepoints up to Week 24(Baseline, Day 2, Weeks 1, 2, 3, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22 and 24)
- Absolute Values of Immunoglobulin (Ig), Immunoglobulin M (IgM), and Immunoglobulin A (IgA) at Baseline and Week 24(Baseline, Week 24)
- Absolute Values of Vitamin B12 at Baseline and Week 24(Baseline, Week 24)
- Absolute Values of Iron and Unsaturated Iron Binding Capacity at Baseline and Week 24(Baseline, Week 24)
- Absolute Values of Vitamin B9 up to Week 24(Baseline up to Week 24)
- Absolute Values of Ferritin at Baseline and Week 24(Baseline, Week 24)
- Absolute Values of Magnesium, Total Cholesterol, and Triglycerides at Week 24(Baseline, Week 24)
- Change From Baseline in Alpha-1 Antitrypsin Levels in Stool at Week 12 and Week 24(Week 12, Week 24)
- Change From Baseline in Alpha-1 Antitrypsin Levels in Blood at Week 12 and Week 24(Week 12, Week 24)
研究者
研究点 (1)
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