NCT02376751No Longer Available不适用
AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- No Longer Available
研究概览
简要总结
This is an open-label, multicenter expanded access protocol to allow patients with a confirmed diagnosis of Lysosomal Acid Lipase (LAL) Deficiency in the United States (US), access to sebelipase alfa (recombinant lysosomal acid lipase [rhLAL]) until commercial product is available.
Patients enrolled in the expanded access protocol will receive 1 mg/kg intravenous infusions of sebelipase alfa every other week.
研究设计
- 研究类型
- Expanded Access
入排标准
- 年龄范围
- 8 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient is ≥ 8 months of age at commencement of treatment with sebelipase alfa.
- •Patient has a confirmed diagnosis of LAL Deficiency.
- •Patient or patient's parent or legal guardian (if applicable) consents to participation in the study. If the patient is of minor age, he/she is willing to provide assent where required per local regulations, and if deemed able to do so.
- •Male and female patients of childbearing potential must use a highly reliable method of birth control (expected failure rate less than 5% per year) from the time they commence treatment through 4 weeks after the last dose of sebelipase alfa.
- •Women of childbearing potential must have a negative serum pregnancy test at commencement of treatment with sebelipase alfa.
排除标准
- •Women who are nursing or pregnant.
- •Patients who received an investigational product within 30 days (for a small molecule) or 60 days (for a biologic) of commencing treatment, and which in the opinion of the investigator or Sponsor, may negatively impact patient safety.
- •Patients who have received sebelipase alfa as part of a clinical trial that is currently active.
- •Patients with known hypersensitivity to eggs.
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