NL-OMON55891招募中3 期
An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia - R1500-CL-1719 (0456/0171)
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 5
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
- 12 至 99（—）
入选标准
- •A patient must meet the following criteria to be eligible for inclusion in the
- •1. Male and female patients >=12 years of age with HoFH. Patients aged >=12
- •years old will be enrolled only in countries where permitted by the Regulatory
- •Agency and Institutional Review Board (IRB) or Ethics Committee (EC).
- •2. Diagnosis of functional HoFH by at least 1 of the following genetic or
- •clinical criteria:
- •a. Documented functional mutation or mutations in both LDLR alleles
- •Note: patients who have null receptor mutations on both LDLR alleles, ie,
- •double null, are eligible
- •b. Presence of homozygous or compound heterozygous mutations in Apo B or PCSK9
- •Note: patients who are double heterozygous, ie, mutations on different genes
- •(eg, LDLR/PCSK9) and patients with homozygous LDLRAP1 mutations are eligible
- •c. Untreated TC >500 mg/dL (12.93 mmol/L) and TG <300 mg/dL (3.39 mmol/L)
- •both parents with documented TC >250 mg/dL (6.47 mmol) OR cutaneous or
- •tendinous xanthoma before the age of 10 years
- •3. For patients who have participated in a previous evinacumab or alirocumab
- •study: completion of the study in which they participated.
- •4. Willing and able to comply with clinic visits and study-related procedures.
- •5. Provide signed informed consent.
排除标准
- •Exclusion Criteria for Evinacumab-Naïve Patients, A patient who meets any of
- •the following criteria will be excluded from the study:
- •1. Concomitant medications and procedures that have not been stable prior to
- •the baseline visit (see Section 7.7.2 for medications and procedures and their
- •associated required duration of therapy).
- •2. Any new condition or worsening of an 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.
- •3. History of a MI, unstable angina leading to hospitalization, coronary artery
- •bypass graft surgery, percutaneous coronary intervention, uncontrolled cardiac
- •arrhythmia, carotid surgery or stenting, stroke, transient ischemic attack,
- •valve replacement surgery, carotid revascularization, endovascular procedure or
- •surgical intervention for peripheral vascular disease within 3 months prior to
- •the baseline visit
- •4. Presence of any clinically significant uncontrolled endocrine disease known
- •to influence serum lipids or lipoproteins
- •Note: patients on thyroid replacement therapy can be included if the dosage of
- •replacement therapy has been stable for at least 12 weeks prior to screening
- •and the thyroid stimulating hormone (TSH) level is within the normal range of
- •the central laboratory at the screening visit
- •5. Newly diagnosed (within 3 months prior to screening visit diabetes mellitus
- •or poorly controlled (HbA1c >9%) diabetes
- •6. Use of systemic corticosteroids, unless used as replacement therapy for
- •pituitary/adrenal disease with a stable regimen for at least 6 weeks prior to
- •screening visit
- •Note: topical, intra-articular, nasal, inhaled and ophthalmic steroid therapies
- •are not considered as *systemic* and are allowed
- •7. Use of estrogen or testosterone therapy unless the regimen has been stable 6
- •weeks prior to the screening visit and no plans to change the regimen during
- •8. Systolic blood pressure >160 mmHg or diastolic blood pressure >100 mmHg at
- •the screening visit
- •9. History of cancer within the past 5 years, except for adequately treated
- •basal cell skin cancer, squamous cell skin cancer, or in situ cervical cancer
- •10. History of New York Heart Association (NYHA) Class IV heart failure within
- •12 months before screening. Please refer to the protocol for exclusion criteria
- •11 - 19. Exclusion Criteria for Patients from a Previous Evinacumab Study, A
- •patient who meets any of the following criteria will be excluded from the study:
- •1. Significant protocol deviation in the previous study based on the
- •investigator*s judgment, such as non-compliance by the patient.
- •2. Concomitant medications and procedures that have not been stable prior to
- •the baseline visit (see Section 7.7.2 for medications and procedures and their
- •associated required duration of therapy).
- •3. Adverse event leading to permanent discontinuation from previous study.
- •4. Any new condition or worsening of an 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. Please
- •refer to the protocol for exclusion criteria 5 - 19.
研究者
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