NCT06449001招募中3 期
A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Change From Baseline in Hemoglobin (Hgb) Concentration at Week 12
研究概览
简要总结
The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of PNH.
- •CS-EVH defined by: Anemia: Hgb ≤ 11.0 g/dL, and absolute reticulocyte count ≥ 100 × 109/L
- •Treated with ravulizumab or eculizumab for at least 12 weeks immediately preceding Day 1, the dose received should be stable during this period, and there should be no anticipated changes in dosage or interval during the first 12 weeks of this study.
- •all participants must be vaccinated against meningococcal infection from serogroups A, C, W, and Y and serogroup B within 3 years prior to, or at least 14 days prior to Day 1
- •vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae
排除标准
- •Platelet count < 30000/μL or there is a need for platelet transfusions.
- •ANC < 500/μL.
- •Clinically significant laboratory abnormalities related to liver function, including:
- •ALT > 2 × ULN or ALT > 3 × ULN for participants with documented liver iron overload defined by serum ferritin values ≥ 500 ng/mL.
- •Direct bilirubin > 2 × ULN, unless, in the Investigator's opinion, is due to hemolysis or Gilbert's syndrome based on medical history.
- •Current evidence of biliary cholestasis.
- •Known aplastic anemia or other bone marrow failure that requires HSCT or other therapies, including anti-thymocyte globulin and immunosuppressants unless the dosage of immunosuppressant has been stable for at least 12 weeks before Day 1 and is expected to remain stable through Week
- •History of a major organ transplant (eg, heart, lung, kidney, liver) or HSCT.
- •Known or suspected complement deficiency.
- •Active bacterial or viral infection, a body temperature > 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to first study intervention administration.
研究组 & 干预措施
Danicopan
Experimental
Participants will receive a 12-week weight-based open-label treatment period and up to 1 year open-label long term extension period.
干预措施: Danicopan (Drug)
结局指标
主要结局
Change From Baseline in Hemoglobin (Hgb) Concentration at Week 12
时间窗: Baseline, Week 12
次要结局
- Number of Participants With Transfusion Avoidance Through Weeks 12 and 24(Weeks 12 and 24)
- Maximum Plasma Concentration (Cmax) of Danicopan(Day 1 up to Week 12)
- Change From Baseline in Absolute Reticulocyte Count at Weeks 12 and 24(Baseline, Weeks 12 and 24)
- Change from Baseline in Pediatric Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Weeks 12 and 24(Baseline, Weeks 12 and 24)
- Change from Baseline in Serum Alternative Pathway (AP) Activity(Baseline up to Week 64)
- Change from Baseline in Plasma Bb Concentrations(Baseline up to Week 64)
- Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Generic Core Scales Score at Weeks 12 and 24(Baseline, Weeks 12 and 24)
- Acceptability and Palatability Questionnaire Score(Week 2)
- Change from Baseline in Hgb Concentration at Week 24(Baseline, Week 24)
研究者
研究点 (1)
Loading locations...
相似试验
已完成
3 期
Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)Paroxysmal Nocturnal HemoglobinuriaNCT04469465Alexion Pharmaceuticals, Inc.86
进行中（未招募）
1 期
Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)MedDRA version: 21.1Level: PTClassification code 10034042Term: Paroxysmal nocturnal haemoglobinuriaSystem Organ Class: 10038359 - Renal and urinary disordersEUCTR2019-003829-18-GBAlexion Pharmaceuticals Inc.84
进行中（未招募）
1 期
Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) (ALPHA)Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)MedDRA version: 21.1Level: PTClassification code 10034042Term: Paroxysmal nocturnal haemoglobinuriaSystem Organ Class: 10038359 - Renal and urinary disordersEUCTR2019-003829-18-GRAlexion Pharmaceuticals Inc.84
招募中
3 期
Pivotal study of danicopan as add-on therapy to a C5 inhibitor (eculizumab or ravulizumab) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have Clinically Evident EVHJPRN-jRCT2021210014Kamata Takeo84
进行中（未招募）
1 期
Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) (ALPHA)EUCTR2019-003829-18-DEAlexion Pharmaceuticals Inc.84