NCT03053102已完成2 期
A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria
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
- Change From Baseline In Serum LDH Levels At Day 28
研究概览
简要总结
The purpose of this study was to determine the safety and efficacy of ACH-0144471 (also known as danicopan and ALXN2040) in currently untreated participants with PNH.
详细描述
After 12 weeks of treatment, participants deriving clinical benefit were offered enrollment in a separate long-term extension study (ACH471-103, NCT03181633).
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Currently untreated PNH participants with PNH Type III erythrocyte and/or granulocyte clone size ≥10% and anemia (hemoglobin <12 grams/deciliter) with adequate reticulocytosis (as determined by the Investigator).
- •LDH ≥1.5 x the upper limit of normal.
- •Platelets ≥50,000/microliter without the need for platelet transfusions.
- •Documentation of vaccination for Neisseria meningitidis, Haemophilus influenza, and Streptococcus pneumoniae, or willingness to receive vaccinations during the screening period.
- •Negative pregnancy test for females prior to dosing and throughout the study.
排除标准
- •History of a major organ transplant (for example, heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplant.
- •Participants who had received another investigational agent within 30 days or 5 half-lives of the investigational agent prior to study entry, whichever is greater.
- •Participants who had received eculizumab at any dose or interval within the past 75 days before study entry.
- •Participants with known or suspected complement deficiency.
- •Participants with active bacterial infection or clinically significant active viral infection, a body temperature >38°Celsius, or other evidence of infection on Day 1, or with a history of febrile illness within 14 days prior to first study drug administration.
- •History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection.
- •Females who were pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration or participants with a female partner who was pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration.
研究组 & 干预措施
Danicopan
Experimental
Starting doses of danicopan ranged from 100 to 150 milligrams (mg) three times daily (TID), with subsequent dose escalation up to 200 mg TID based on response (clinical and biochemical) for 28 days (Part 1). Participants with reductions in lactate dehydrogenase (LDH) meeting specified criteria were offered continued dosing beyond Day 28, for up to 8 additional weeks (Part 2).
干预措施: Danicopan (Drug)
结局指标
主要结局
Change From Baseline In Serum LDH Levels At Day 28
时间窗: Baseline, Day 28
Change from Baseline = Serum LDH levels on Day 28 - Baseline Serum LDH levels.
次要结局
- Change From Baseline In Hemoglobin (Hgb) At Days 28 And 84(Baseline, Days 28 and 84)
- Change From Baseline In Serum LDH Levels At Day 84(Baseline, Day 84)
- Paroxysmal Nocturnal Hemoglobinuria (PNH) Type III Red Blood Cell (RBC) Clone Size(Baseline, Day 28, and Day 84)
- Serious Adverse Events (SAEs), Grade 3 And Grade 4 Treatment-emergent Adverse Events (TEAEs), And Adverse Events (AEs) Leading To Discontinuation(After the first dose of study medication (Day 1) through 14 days after the last dose of study drug (up to Day 104))
- Grade 3 And Grade 4 Laboratory Abnormalities(After the first dose of study medication (Day 1) through 14 days after the last dose of study drug (up to Day 104).)
- Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve From Time Of Administration To 8 Hours Post-dose (AUC0-8)(Days 6 and 20)
- PK: Maximum Plasma Concentration (Cmax)(Days 6 and 20)
- PK: Time To Maximum Concentration (Tmax)(Days 6 and 20)
- Complement Alternative Pathway (AP) Functional Activity(Baseline and Day 28)
- Complement Bb(Baseline and Day 28)
研究者
研究点 (1)
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