EUCTR2011-002880-42-GB进行中（未招募）不适用
An Open Label Multicenter Extension Study to Evaluate the Long-Term Efficacy and Safety of SBC 102 in Children with Lysosomal Acid Lipase Deficiency Who Previously Received Treatment with SBC-102 - Extension to LAL-CL03 in children with LAL 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 of study treatment and procedures, and provides written informed consent/permission prior to any study procedures being performed.
- •2. Subject completed treatment in study LAL-CL03.
- •Subject received treatment with SBC-102 for at least 4 months under an expanded access treatment regimen,
- •3. Subject had no life-threatening or unmanageable study drug toxicity during treatment with SBC-102 under study LAL-CL03 or an expanded access treatment regimen.
- •4. Subjects must be <12 months of age unless previously enrolled in study LAL-CL03.
- •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 0
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •1. Clinically important concurrent disease including, but not restricted to, congestive heart failure, acute or chronic renal failure, additional severe congenital abnormality, or other extenuating circumstances, including life threatening undernutrition or rapidly progressing liver disease, that in the opinion of the investigator would interfere with study participation.
- •2. Myeloablative preparation, or other systemic pre-transplant conditioning, for haematopoietic stem cell or liver transplantation.
- •3. Previous haematopoietic stem cell transplant.
研究者
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