An Open-Label Study of Volanesorsen Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)

Akcea Therapeutics0 个研究点目标入组 70 人开始时间: 2016年2月26日最近更新: 2020年7月13日

适应症

试验速览

暂无简介。

•1. Able and willing to participate in a 65-week study
•2. Group 1 and 2: Satisfactory completion of ISIS 304801-CS6 or ISIS 304801-
•CS16 (index studies) with an acceptable safety profile, per
•Sponsor and Investigator judgment
•3. Group 3: Patients who did not participate in the CS6 or CS16 index
•studies and meet additional inclusion criteria of Familial
•Chylomicronemia Syndrome (FCS) may enroll in the study.
•3a. History of chylomicronemia
•3b. A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia)
•3c. Fasting triglycerides = 750 mg/dL (8.4 mmol/L) at Screening
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 70
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•For Group 1 and Group 2:
•1. Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study
•2. Unwilling to comply with lifestyle requirements for the duration of the study
•For Group 3:
•a. Diabetes mellitus if newly diagnosed or if HbA1c = 9.0%
•b. Active pancreatitis within 4 weeks of screening
•c. Acute Coronary Syndrome within 6 months of screening
•d. Major surgery within 3 months of screening
•e. Treatment with Glybera therapy within 2 years of screening
•f. Have any other conditions in the opinion of the investigator which could interfere with the patient participating in or completing the study.

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