NCT03544060No Longer Available不适用
Volanesorsen (ISIS 304801) Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- No Longer Available
研究概览
简要总结
The purpose of this program is to provide expanded access to volanesorsen for up to 100 Patients with Familial Chylomicronemia Syndrome (FCS).
详细描述
The Program is intended to provide expanded access to volanesorsen for eligible patients with FCS who have limited or no available treatment options.
研究设计
- 研究类型
- Expanded Access
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
- •Patients who participated in the APPROACH Open Label trial, must have completed the open label extension trial for volanesorsen for a length of time consistent with the study protocol (≥ 1 year). Patients in the APPROACH open Label trial for less than a year require approval from Akcea to participate in the EAP. Patients who did not participate in the APPROACH open label trial require approval from Akcea for entry into EAP.
- •Patients not participating in the APPROACH open label trial must have a diagnosis of FCS as determined by the participating physician, as outlined in the Volanesorsen EAP protocol. Akcea will review each application to determine eligibility.
- •Male patients and female patients of childbearing potential must continue to use appropriate contraception with their partners, or refrain from sexual activity
排除标准
- •Patients who have any new conditions or worsening of existing conditions which in the opinion of the Physician would make the patient unsuitable for treatment with volanesorsen.
- •Volanesorsen naïve patients with baseline platelet values ≤ 140,000/mm3
- •Patients not willing to adhere to mandatory blood draws for platelet monitoring
- •Patients who in the opinion of Akcea's medical team, are not eligible candidates for volanesorsen therapy.
- •Any patient who plans to or becomes pregnant.
- •Any patient who was withdrawn from the APPROACH open label study due to a serious adverse event related to volanesorsen therapy.
研究者
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