EUCTR2020-002997-28-NL进行中（未招募）1 期
An Open-Label Study of Volanesorsen (ISIS 304801) AdministeredSubcutaneously to Pediatric Patients with Familial ChylomicronemiaSyndrome (FCS)
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 16
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Cohort 1 Inclusion Criteria:
- •1. Age = 12 years and < 18 years at time of informed consent and assent, as required by regional laws or regulations
- •Cohort 2 Inclusion Criteria:
- •1. Age = 2 years to < 12 years at time of informed consent and assent, as required by regional laws or regulations
- •Cohort 1 and 2 Inclusion Criteria:
- •2. Written informed consent/assent and any authorizations required by law must be obtained from the patient and parent(s) (or legally authorized representative) after the nature of the study has been explained and before any assessment is performed
- •3. Confirmed homozygote, compound heterozygote or double heterozygote for known loss-of-function mutations in FCS-causing genes (such as LPL, APOC2, APOA5, LMF1 or GP1HBP1)
- •4. Fasting TG = 880 mg/dL (~10 mmol/L) at screening. If the fasting TG is < 880 mg/dL, up to two additional tests may be performed in order to qualify
- •5. Willing to follow a diet for optimal control of TGs and disease management during the study
- •For patients of reproductive age, an additional inclusion criterion:
- •6. Satisfy one of the following:
- •a. Females: Non-pregnant and non-lactating; abstinent*, or if engaged in sexual relations of childbearing potential, patient agrees to use an acceptable contraceptive method (refer to Section 6.3.1) from time of signing the informed consent/assent form until 13 weeks after the last visit in the Treatment Period
- •b. Males: Abstinent* or if engaged in sexual relations with a female of childbearing potential, patient is utilizing an acceptable contraceptive method (refer to Section 6.3.1) from the time of signing the informed consent/assent form until 13 weeks after the last visit in the Treatment Period
- •* Abstinence (i.e. refraining from heterosexual intercourse) is only acceptable to meet the contraception requirements of the study if it is true abstinence. This means that abstinence must be the preferred and usual lifestyle of the patient. Periodic abstinence (i.e. calendar, ovulation, symptothermal, post-ovulation methods), declaration of abstinence for the duration of a trial, and/or withdrawal are not acceptable methods of
- •contraception
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 16
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Body weight < 10 kg
- •2. Type 1 or Type 2 diabetes mellitus
- •3. Any of the following laboratory values at screening
- •a. Hepatic:
- •Total bilirubin > upper limit of normal (ULN), unless prior diagnosis and documentation of
- •Gilbert’s syndrome in which case total bilirubin must be = 3 mg/dL
- •Alanine Aminotransferase (ALT) > 2.0 x ULN
- •Aspartate Aminotransferase (AST) > 2.0 x ULN
- •Persistently positive proteinuria: urine protein/creatinine ratio (UPCR) > 0.2 mg/mg on 2 tests
- •separated by at least 7 days
- •Persistently positive hematuria: urine microscopy showing > 5 red blood cells per high power field
- •on 2 tests separated by at least 7 days
- •Estimated GFR (eGFR) < 75 mL/min/1.73m2 using the Bedside Schwartz formula (Selistre 2016)
- •c. Low density lipoprotein-cholesterol (LDL-C) > 130 mg/dL at screening
- •d. Platelet count < 140,000/mm3
- •e. Any other laboratory abnormalities which, in the opinion of the Investigator or the Sponsor, would
- •make the patient unsuitable for inclusion
- •4. History of chronic kidney disease
- •5. Uncontrolled thyroid disease
- •6. Has uncontrolled hypertension (average clinic measured SBP and/or DBP =95th percentile on the basis of
- •age, sex, and height percentiles)**
- •**See Appendix E Blood Pressure Tables for Boys and Girls by Age and Height Percentile
- •7. History of bleeding diathesis or coagulopathy or clinically-significant abnormality in coagulation parameters
- •at screening
- •8. Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to
- •Study Day 1
- •9. Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis
- •10. History of malignancy
- •11. History of alcoholism or drug/chemical abuse within 1 year of screening
- •12. Treatment with another investigational drug, biological agent, or device within one month of screening, or 5 half-lives of investigational agent, whichever is longer
- •13. Unwilling or unable to comply with lifestyle requirements
- •14. History of major psychosis or major depressive disorder
- •15. Use of any of the following:
- •a. Statins, omega-3 fatty acids (prescription and over-the-counter [OTC]), or fibrates unless on a stable dose for at least 3 months prior to screening and dose and regimen expected to remain constant during the Treatment Period. Patients taking OTC omega-3 fatty acids should make every effort to remain on the same brand throughout the study
- •b. Nicotinic acid or derivatives of nicotinic acid within 4 weeks prior to screening
- •c. Systemic corticosteroids or anabolic steroids within 6 weeks prior to screening
- •d. Treatment with oral anticoagulant medications
- •e. Estrogens or progestins (including birth control pills) unless on a stable dose for at least 6 weeks prior to screening and dose and regimen expected to remain constant during the Treatment Period
- •f. Plasma apheresis within 4 weeks prior to screening or planned during the study
- •g. Prior exposure to volanesorsen within the last 6 months
- •h. Any other medication unless stable at least 4 weeks prior to screening. Occasional or intermittent use of over-the-counter medications will be allowed at Investigator’s discretion
- •16. Known hypersensitivity to any of the excipients of volanesorsen
- •17. In the opinion of the Investigator, inability to comply with the protocol
- •18. Have any other conditions, including history of significant cardiac disease which, in the opinion of the Investigator or the Sponsor wo
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