NCT07187401招募中1 期
A Randomized, Double-Blind, Placebo-Controlled, First-In-Human Study of the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of ALN-CFB, an Investigational siRNA Therapeutic Against Complement Factor B, in Participants With Paroxysmal Nocturnal Hemoglobinuria and Persistent Anemia on Approved C5-Inhibitor Therapy
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 24
- 试验地点
- 8
- 主要终点
- Occurrence of Treatment-Emergent Adverse Events (TEAEs)
研究概览
简要总结
This study is researching an experimental drug called ALN-CFB. The study is focused on people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are currently taking a complement component C5 inhibitor ("C5-inhibitor") and continue to have anemia (low red blood cell count).
The aim of the study is to see how tolerable ALN-CFB is compared to placebo. A placebo looks like the study drug but does not contain any drug.
The study is looking at several other research questions, including:
- What side effects may happen from taking ALN-CFB
- How much ALN-CFB is in the blood at different times
- How much Complement Factor B (CFB) protein levels in the blood are affected by ALN-CFB
详细描述
The protocol will be amended to describe Part B of the study after Part A data have been analyzed.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Has been diagnosed with PNH confirmed by a history of high flow cytometry from prior testing
- •Treated with a stable dose of C5 inhibitor (eculizumab or approved eculizumab biosimilar, ravulizumab, or crovalimab) for at least 24 weeks prior to screening visit, as described in the protocol
- •Has hemoglobin ≤10.5 g/dL at screening visit 1, with evidence of anemia prior to this visit, as described in the protocol
- •Has peripheral blood reticulocyte count of ≥100 x 10^9/L at screening visit 1
排除标准
- •Has history of bone marrow transplantation or receipt of an organ transplant
- •Has history of meningococcal infection or similar recurrent infections by other encapsulated bacterial organisms
- •Has any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period
- •Has laboratory evidence of bone marrow failure, as described in the protocol
- •Have recent, unstable medical conditions, not related to PNH or PNH-related complications, as described in the protocol
- •NOTE: Other Protocol Defined Inclusion / Exclusion Criteria Apply
研究组 & 干预措施
Single-Ascending Dose Escalation
Experimental
干预措施: ALN-CFB (Drug)
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
结局指标
主要结局
Occurrence of Treatment-Emergent Adverse Events (TEAEs)
时间窗: Through 365 Days
Severity of TEAEs
时间窗: Through 365 Days
次要结局
- Concentrations of combined ALN-CFB and major metabolites in plasma(Through 365 Days)
- Concentrations of combined ALN-CFB and major metabolites in urine(Through 24 Hours following ALN-CFB administration)
- Absolute change from baseline in CFB concentration(Baseline, Through 365 Days)
- Percentage change from baseline in CFB concentration(Baseline, Through 365 Days)
研究者
研究点 (8)
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