EUCTR2011-000032-28-IE进行中（未招募）1 期
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 D
适应症
试验速览
- 阶段
- 1 期
- 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
- •A subject must meet all of the following criteria to be eligible to
- •participate in this study:
- •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
- •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 by:
- •weight decreasing across at least 2 of the 11 major centiles on a
- •standard World Health Organization (WHO) weight-for-age (WFA)
- •chart (1st, 3rd, 5th, 10th, 25th, 50th, 75th, 90th, 95th, 97th, 99th);
- •body weight in kg below the 10th centile on a standard WHO WFA
- •chart AND no weight gain for the 2 weeks prior to screening;
- •loss of >5% of birth weight in a child who is older than 2 weeks of
- •*NOTE regarding Inclusion Criterion # 3: In the unusual circumstance
- •where a child has a rapidly progressive course of LAL Deficiency but
- •does not meet growth failure criteria as defined above, the child may be
- •included in the study if the Investigator has substantial clinical concerns
- •based on evidence of the rapid disease progression requiring urgent
- •medical intervention. Inclusion under these exceptional circumstances
- •requires submission of a written summary of the infant’s medical status
- •for review by the Sponsor and must be approved by a written
- •confirmation from the Sponsor after consultation with the Safety
- •Committee (SC). The subject must meet all other entry criteria as stated.
- •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 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 analysis.)
- •3. Has received an investigational medicinal product other than sebelipase alfa within 14 days prior to the first dose of sebelipase alfa 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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