NL-OMON53717招募中2 期
An Open-Label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Bempedoic Acid in Pediatric Patients (6 to 17 Years of Age) with Heterozygous Familial Hypercholesterolemia - Esperion 1002-041
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 15
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- 2 至 17（—）
入选标准
- •1. The patient*s parent(s)/guardian(s) must be willing to provide written
- •informed consent and the patient must provide informed assent before any
- •study-specific procedures are performed;
- •2. The patient must be aged 6-17 years old;
- •3. The patient must weigh at least 16 kg;
- •4. The patient must have a diagnosis of HeFH per MEDPED criteria by meeting at
- •least one of the following clinical criteria*
- •a. Documented diagnosis of HeFH determined by positive genetic testing; or
- •b. Documented LDL-C or TC meeting one or more of the following criteria:
- •i. LDL-C >200 mg/dL (5.2 mmol/L) or total cholesterol (TC) >270 mg/dL (7.0
- •mmol/L), with no first- second- or third-degree relative with documented FH
- •diagnosis (general population); or
- •ii. LDL-C >155 mg/dL (4.0 mmol/L) or TC >220 mg/dL (5.7 mmol/L), and also
- •having a first-degree relative with documented familial hypercholesterolemia
- •(FH) diagnosis; or
- •iii. LDL-C >165 mg/dL (4.3 mmol/L) or TC >230 mg/dL (5.9 mmol/L), and also
- •having a second-degree relative with documented FH diagnosis; or
- •iv. LDL-C >170 mg/dL (4.4 mmol/L) or TC >240 mg/dL (6.2 mmol/L), and also
- •having a third-degree relative with documented FH diagnosis
- •5. Current treatment with approved stable LMTs, including optimal dose of
- •statin +/- other LMT(s), at stable dose for at least 4 weeks prior to screening
- •Visit S1 (6 weeks for fibrates; however, gemfibrozil is not allowed in patients
- •taking a statin as per co-administration instructions defined in the statin
- •label) and must remain on that stable dose throughout the duration of the
- •6. The patient must have a fasting LDL-C level >=130 mg/dL (3.4 mmol/L);
- •7. The patient may be male or female. Females must not be pregnant (or planning
- •to become pregnant within 30 days after the last dose of investigational
- •medicinal product [IMP]) breastfeeding and must be sexually inactive or willing
- •to use 1 acceptable method of birth control. The minimal requirement for use
- •of acceptable contraception is from the time the informed consent form (ICF) is
- •signed, during the study period, and for at least 30 days after the last dose
排除标准
- •1. The patient has a diagnosis of HoFH or compound HeFH;
- •2. The patient has a fasting triglyceride (TG) level >=400 mg/dL (4.5 mmol/L);
- •3. The patient has uncontrolled hypothyroidism, including a value for
- •thyroid-stimulating hormone (TSH) < lower limit of normal (LLN) or >1.5 × the
- •upper limit of normal (ULN);
- •4. The patient has liver disease or dysfunction, including:
- •a. positive serology for hepatitis B surface antigen (HBsAg) and/or hepatitis C
- •virus antibodies (HCV-AB), or
- •b. serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST)
- •value >=2 × ULN and/or serum total bilirubin (TB) value >=2 × ULN. If the serum
- •TB value is >=1.2 × ULN, a reflex indirect (unconjugated) bilirubin will be
- •obtained and, if consistent with Gilbert*s disease or if the patient has a
- •history of Gilbert*s disease, the patient may be enrolled in the study.
- •5. The patient has renal dysfunction or glomerulonephritis, including an
- •estimated glomerular filtration rate (eGFR) <75 mL/min/1.73 m2 (as determined
- •by the central laboratory using the Revised [*Bedside*] Schwartz formula);
- •6. The patient has Stage 2 hypertension (based on gender, age and height;
- •7. The patient has a gastrointestinal condition that may affect drug
- •absorption;
- •8. The patient has a history of hematologic or coagulation disorders, anemia,
- •or a hemoglobin (Hgb) level <11.5 g/dL;
- •9. The patient has type 1 or type 2 diabetes, or newly diagnosed impaired
- •glucose tolerance (within 3 months of Screening);
- •10. The patient had an active malignancy, including those requiring surgery,
- •chemotherapy, and/or radiation, in the past 5 years. Nonmetastatic basal or
- •squamous cell carcinoma of
- •the skin and cervical carcinoma in situ are allowed;
- •11. The patient has an unexplained (ie, not associated with recent trauma or
- •physically strenuous activity) serum creatine kinase (CK) value >3 × ULN at any
- •time before randomization. Patients with an explained elevation in serum CK
- •must have single repeat serum CK value <=3 × ULN before enrollment;
- •12. The patient has a history of drug or alcohol abuse within the last 2 years
- •or is unwilling to refrain from alcohol consumption for the duration of the
- •study, or uses any illicit drugs, or has a history of amphetamine or
- •derivatives abuse or cocaine abuse. Patients who are using amphetamine
- •derivatives prescribed by and who are under the care of a health care
- •practitioner can be enrolled after evaluation by the Investigator;
- •13. The patient has donated blood, undergone multiple blood draws in a clinical
- •study, experienced major trauma, received a blood transfusion, or undergone
- •surgery, with or without blood loss, within 30 days before enrollment;
- •14. The patient has used any experimental or investigational drugs within 30
- •days before screening and throughout the trial;
- •15. The patient has previously participated in a clinical study of bempedoic
- •16. The patient is taking any of the following medications or therapies, except
- •as indicated below:
- •a. Mipomersen or lomitapide (current or within 6 months of Screening).
- •b. PCKS9 inhibitors including evolocumab or alirocumab (current or within 3
- •months of Screening).
- •c. Lipid apheresis (current or within 8 weeks of Screening or intends to have
- •lipid apheresis treatments throughout the trial).
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研究者
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