EUCTR2011-002880-42-FR进行中（未招募）1 期
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 - SBC-102 in Children with Growth Failure Due to Lysosomal Acid Lipase Deficiency
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 6
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
- •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 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 hematopoietic stem cell or liver transplantation.
- •3.Previous hematopoietic stem cell transplant.
研究者
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A research study to look at the safety, effectiveness and the long-term effects on the body of a new drug, SBC-102, in children with growth problems caused by a deficiency in the enzyme that breaks down fats who were previously treated with SBC-102Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease).MedDRA version: 14.0Level: SOCClassification code 10027433Term: Metabolism and nutrition disordersSystem Organ Class: 10027433 - Metabolism and nutrition disordersMedDRA version: 14.0Level: HLGTClassification code 10021605Term: Inborn errors of metabolismSystem Organ Class: 10027433 - Metabolism and nutrition disordersMedDRA version: 14.0Level: HLTClassification code 10024579Term: Lysosomal storage disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2011-002880-42-GBSynageva Biopharma Corp.10
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