A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 11
- 试验地点
- 25
- 主要终点
- Risdiplam Free Fraction
研究概览
简要总结
This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
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
- — 至 19 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female newborn infant aged <20 days at first dose
- •Newborn infants with genetic diagnosis of 5q-autosomal recessive SMA or newborn infants identified as positive for SMA via newborn screening or via prenatal testing.
- •Gestational age equal to or greater than 37 weeks
- •Receiving adequate nutrition and hydration at the time of screening
- •Adequately recovered from any acute illness at baseline and considered well enough to participate in the study
- •Parent/caregiver is willing to consider nasogastric, nasojejunal, or gastrostomy tube placement during the study to maintain safe hydration, nutrition, and treatment delivery, if recommended by the investigator.
排除标准
- •Presence of clinical symptoms or signs consistent with SMA Type 0
- •In the opinion of the investigator, inadequate venous or capillary blood access for the study procedures
- •Systolic blood pressure or diastolic blood pressure or heart rate abnormalities
- •Presence of clinically relevant electrocardiogram (ECG) abnormalities
- •The infant (or the person breastfeeding the infant) taking any of the following: any inhibitor of CYP3A4 taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing, any inducer of CYP3A4 taken within 4 weeks (or within 5 times the elimination half-life, whichever is longer prior to dosing, and/or use of any multidrug and toxin extrusion (MATE) substrates taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing
- •Concurrent or previous administration of nusinersen or onasemnogene abeparvovec
- •Clinically significant abnormalities in laboratory test
研究组 & 干预措施
Risdiplam
Participants will receive risdiplam once daily for 28 days.
干预措施: Risdiplam (Drug)
结局指标
主要结局
Risdiplam Free Fraction
时间窗: From Day 1 through Day 28
Plasma Concentrations of Risdiplam
时间窗: From Day 1 through Day 28
Area Under the Plasma Concentration-Time Curve (AUC) of Risdiplam
时间窗: From Day 1 through Day 28
Steady-state Concentration (Css) of Risdiplam
时间窗: From Day 1 through Day 28
Percentage of Participants With Adverse Events
时间窗: Up to 30 days after the final dose of study treatment (up to 58 days)
Percentage of Participants With Serious Adverse Events
时间窗: Up to 30 days after the final dose of study treatment (up to 58 days)
Percentage of Participants With Treatment Discontinuation due to Adverse Events
时间窗: Up to 30 days after the final dose of study treatment (up to 58 days)
次要结局
未报告次要终点