NL-OMON46961已完成3 期
An Open-Label Study of Volanesorsen Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS) - ISIS-CS7 study/ APPROACH Open Label
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •1. Must give written informed consent to participate in the study (signed and dated) and any authorizations required by law
- •2. Age >= 18 years at time of informed consent
- •3. 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 judgement. ;Group 2: Patients who enrolled in ISIS 304801-CS16 must also meet the following criteria in order to enter into the open-label Study:
- •a. History of chylomicronemia as evidenced by documentation of lactescent serum (a creamy top layer after ultracentrifugation of a fasting blood sample) or documentation of fasting TG measurement >= 880 mg/dL (10 mmol/L)
- •b. A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia) by documentation of at least one (1) of the following:
- •Confirmed homozygote, compound heterozygote or double heterozygote for known loss-of-function mutations in Type 1-causing genes (such as LPL, apoC-II, GPIHBP1, or LMF1)
- •Post heparin plasma LPL activity of <= 20% of normal in medical history. Note: testing of LPL activity should not be performed to confirm eligibility for the study
- •c. Group 2: Fasting TG >= 750 mg/dL (8.4 mmol/L) at Qualification for the ISIS 304801-CS16 study
- •4. Able and willing to participate in a 65-week study
- •5. Satisfy one (1) of the following:
- •a. Females: Non-pregnant and non-lactating; surgically sterile (e.g., tubal occlusion, hysterectomy, bilateral salpingectomy, bilateral oophorectomy), post-menopausal (defined as 12 months of spontaneous amenorrhea in females > 55 years of age or, in females <= 55 years, 12 months of spontaneous amenorrhea without an alternative medical cause and FSH levels in the postmenopausal range for the laboratory involved), abstinent*, or if engaged in sexual relations of child-bearing potential, patient is using an acceptable contraceptive method (refer to Section 6.3.1) from time of signing the informed consent form until 13 weeks after the last dose of Study Drug administration.
- •b. Males: Surgically sterile, abstinent* or if engaged in sexual relations with a female of child-bearing potential, patient is utilizing an acceptable contraceptive method (refer to Section 6.3.1) from the time of signing the informed consent form until 13 weeks after the last dose of Study Drug administration.
- •* Abstinence is only acceptable as true abstinence, i.e., when this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods), declaration of abstinence for the duration of a trial and withdrawal are not acceptable methods of contraception.
排除标准
- •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
- •Unwilling to comply with lifestyle requirements for the duration of the study (protocol section 6.3)
研究者
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