EUCTR2010-024068-16-CZ进行中（未招募）不适用
An open label multicenter study to evaluate the safety, tolerability and pharmacokinetics of SBC-102 in adult patients with liver dysfunction due to lysosomal acid lipase deficiency. - SBC-102 in patients with liver dysfunction due to LAL deficiency
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 9
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. Patient understands the full nature and purpose of the study, including possible risks and side effects, and is willing and able to comply with all study procedures and provide informed consent
- •2. Male or female patients = 18 years = 65 years of age
- •3. Documented decreased LAL activity relative to the normal range of the lab performing the assay or documented result of molecular genetic testing confirming diagnosis of LAL deficiency
- •4. Evidence of liver involvement based on clinical presentation and/or laboratory test results (ALT or AST = 1.5xULN)
- •5. If on a statin or ezetimibe, must be on a stable dose for at least 4 weeks prior to screening
- •6. All women must have negative serum pregnancy test at screening and cannot be breast feeding
- •7. Female subjects of childbearing potential must agree to use a highly effective and approved contraceptive method(s) for the duration of the study and continue to use for 30 days after last dose of study drug. A highly effective method of contraception is defined as:
- •a. strict abstinence;
- •b. bilateral tubal ligation;
- •c. combined oral contraceptives (estrogens and progesterone), implanted or injectable contraceptives on a stable dose for at least 1 month prior to the Screening visit;
- •d. hormonal intra uterine device (IUD) inserted at least 1 month prior to the Screening visit;
- •e. vasectomized partner for at least 3 months prior to the Screening visit.
- •Male subjects, and their partners must be using, and continue to use for 30 days after last dose of study drug, an acceptable method of birth control. Women considered not of childbearing potential must be surgically sterile (total hysterectomy, bilateral salpingo-oophorectomy) or post-menopausal, which is defined as a complete cessation of menstruation for at least one year after the age of 45 years.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range: 0
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 9
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Clinically significant concurrent disease, serious inter-current illness, concomitant medications or other extenuating circumstances that, in the opinion of the Investigator, would either interfere with study participation or the interpretation of the effects of SBC-102.
- •2. Clinically significant abnormal values on laboratory screening tests, other than liver function or lipid panel tests. Subjects with an abnormal laboratory value that is of borderline significance may be allowed to undergo repeat testing once within a 30 day period.
- •3. Patient has participated in a study employing an investigational drug within 30 days of the screening
- •4. Child-Pugh Class C or AST and/or ALT persistently elevated > 3xULN at screening (2 or more occasions)
- •5. Previous hematopoietic bone marrow or liver transplant
- •6. Patient has received prior treatment with enzyme replacement therapy
- •7. Subject has a total score of 8 or more on a screening Alcohol Use Disorders Identification Test (AUDIT)
- •8. Patients with known hypersensitivity to eggs
研究者
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