EUCTR2014-003806-33-GR进行中（未招募）1 期
A Phase 3, Single-arm, Open-label, International, Multi-center Study to Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients with Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 45
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
- •1. Male or female aged =5 and<18 years with diagnosed functional HoFH by at least one of the following criteria.
- •a) Documented functional mutation(s) in both LDL-R alleles or alleles known to affect LDL-R functionality (e.g., apo B defective mutations),
- •b) Skin fibroblast LDL-R activity <20% normal,
- •c) Untreated TC >500 mg/dL (13.0 mmol/L) AND TG <300 mg/dL (3.4 mmol/L) with both parents having documented untreated TC >250 mg/dL (6.5 mmol/L),
- •d) Average fasting LDL-C >300 mg/dL (7.8 mmol/L) on maximally
- •tolerated lipid-lowering therapy as decided by the treating physician with both parents having documented untreated TC >250 mg/dL (6.5 mmol/L).
- •2. Baseline LDL-C >160 mg/dL (4.1 mmol/L) on background therapy and no documented CVD or baseline LDL-C >130 mg/dL (3.4 mmol/L) on background therapy for patients with established CVD (defined as aortic valve disease and/or coronary atherosclerosis).
- •3. Parent/guardian is able to provide written informed consent for patient (according to local legal requirements) prior to any trial procedures.
- •4. Patient must provide informed assent or informed consent according to local legal requirements and must understand the trial procedures and that he or she can withdraw from the study at any time.
- •5. Patient must weigh at least 15 kg and be at or above the 10th percentile in BMI and at least 10th percentile in height for age and gender based on the CDC growth charts.
- •6. Patient and parent/guardian understand and agree that lipid-lowering therapy, including LDL apheresis, must be stable for at least 12 weeks prior to Day 1 of treatment and remain stable through the Week 24 visit of the study.
- •7. Patient must be in stable physical and mental health at Screening.
- •8. Patient/guardian must agree to be compliant with a low-fat diet supplying <20% of energy (calories) from fat, starting at the beginning of the Run-in Period and continuing until completion in the trial.
- •9. Patient/guardian must agree to be compliant with the dietary supplement regimen starting at Week 2 of the Run-in Period and continuing until completion in the trial.
- •10. All female patients of potential child-bearing age and who have attained menarche must have a negative pregnancy test at Screening and during the study.
- •11. Potentially sexually active female patients who are of childbearing age must either be sexually abstinent or follow two acceptable methods of contraception including one highly effective and one effective method (i.e., the use of a hormonal contraceptive in addition to the use of a barrier method plus spermicide) during the study and for 4 weeks after the last dose of study medication. Patients taking estrogen-based oral contraceptives should be advised about possible loss of effectiveness due to diarrhea and/or vomiting. Additional contraceptive measures should be used for 7 days after resolution of symptoms.
- •- Highly effective methods of contraception include: hormonal contraceptives (e.g., combined oral contraceptives, patch, vaginal ring, injectables, and implants); intrauterine device (IUD) or intrauterine system (IUS); vasectomy or tubal ligation.
- •- Effective methods of contraception include: barrier method (e.g., male condom, female condom, cervical cap with spermicide, diaphragm with spermicide, contraceptive sponge).
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 45
- •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)
排除标准
- •1. Other forms of primary hyperlipoproteinemia and secondary causes of hypercholesterolemia (e.g., nephrotic syndrome, hypothyroidism).
- •2. Abnormal liver function test (LFT) at Screening (aspartate aminotransferase [AST] or alanine aminotransferase [ALT] >1.5 times the upper limit of normal [× ULN] and/or total bilirubin >1.5 × ULN in the absence of Gilbert’s syndrome, or alkaline phosphatase [ALP] >1.5 × ULN based on appropriate age and gender normal values).
- •3. Moderate or severe hepatic impairment or active liver disease.
- •4. Serum creatine phosphokinase (CPK) level >2 × ULN.
- •5. Chronic renal insufficiency with glomerular filtration rate (GFR) <70 mL/min calculated using the Schwartz formula.
- •6. History of drug abuse within the last 3 years or habitual alcohol consumption (defined as >1 ounce [28 grams] of liquor or 4 ounce glass [113 grams] of wine, or the equivalent, 3 or more times per week).
- •7. New York Heart Association (NYHA) Class III or IV congestive heart failure.
- •8. Uncontrolled hypertension (defined as mean systolic or diastolic blood pressure >95% of the ULN for age and sex) despite medical therapy.
- •9. In the judgment of the PI, precocious or delayed puberty or endocrine disorder that would affect growth (e.g., hypothyroidism, premature adrenarche).
- •10. History of non-skin malignancy (with the exception of treated basal cell or squamous cell cancer or noninvasive cervical cancer in situ) or other cancers occurring within the past 3 years.
- •11. History of inflammatory bowel disease or other malabsorption syndrome or a history of bowel resection, gastric bypass, or other weight loss surgical procedure.
- •12. Use of mipomersen within 6 months of Screening
- •13. Any medical condition for which the life expectancy is predicted to be less than 5 years.
- •14. Any patient who is unable to avoid treatment with strong or moderate cytochrome P450 3A4 (CYP3A4) inhibitors, or other drugs contraindicated for use with lomitapide during the study.
- •15. Participation in an interventional clinical study within 6 weeks for a statin therapy or within 6 months for any other unapproved therapy.
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