EUCTR2011-000032-28-IT进行中（未招募）不适用
AN OPEN LABEL, MULTICENTER, DOSE ESCALATION STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SBC-102 IN CHILDREN WITH GROWTH FAILURE DUE TO LYSOSOMAL ACID LIPASE DEFICIENCY - SBC-102 in Children with Growth Failure due to LYSOSOMAL ACID LIPASE DEFICIENCY
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 10
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. Subject's parent or legal guardian understands the full nature and
- •purpose of the study, including possible risks and side effects, and
- •provides written informed consent/permission prior to any study
- •procedures being performed
- •2. Male or female child with a documented decreased LAL activity
- •XML File Identifier: xHVSKdDb30v7HfyciGRPYEL0jt0=
- •relative to the normal range of the lab performing the assay or
- •documented result of molecular genetic testing (2 mutations) confirming
- •a diagnosis of LAL Deficiency
- •3. Growth failure with onset before 6 months of age, as defined in the
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 10
- •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. Clinically important concurrent disease or co-morbidities which, in the
- •opinion of the Investigator and Sponsor, would interfere with study
- •participation, including, but not restricted to, congestive heart failure,
- •ongoing circulatory collapse requiring inotropic support, acute or chronic
- •renal failure, additional severe congenital abnormality, or other
- •extenuating circumstances such as life-threatening under nutrition or
- •rapidly progressive liver disease.
- •2. Subject is >24 months of age. (Note: Subjects >8 months of age on
- •the date of first infusion will not be eligible for the primary efficacy
- •3. Has received an investigational medicinal product other than SBC-102
- •within 14 days prior to the first dose of SBC-102 in this study.
- •4. Myeloablative preparation, or other systemic pre-transplant
- •conditioning, for hematopoietic stem cell or liver transplantation.
- •5. Previous hematopoietic stem cell or liver transplant.
- •6. Known hypersensitivity to eggs.
研究者
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