NL-OMON47132已完成不适用
A Prospective and Retrospective Data Collection Study to Evaluate Outcomes in males <= 17 years of age undergoing Allogeneic Hematopoietic Stem Cell Transplantation for the Treatment of Cerebral Adrenoleukodystrophy. - ALD-103
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- bluebird bio
- 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
- Observational
入排标准
- 年龄范围
- 2 至 17（—）
入选标准
- •Subjects must:
- •1. Provide informed consent from a competent custodial parents or guardian with legal capacity to execute a local Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved consent. In addition, informed assent will be sought from capable subjects, in accordance with the directive of the IRB/IEC and with local requirements.
- •2. Be male and <=17 years of age at the time of treatment, for retrospective and partial prospective/retrospective subjects, or at time of parental/guardian consent and, where appropriate, subject assent.
- •3. Have a confirmed diagnosis of CALD as defined by abnormal VLCFA profile and cerebral lesion on brain MRI.
- •4. Depending on the cohort, the subject must:
- •- Be scheduled for allo-HSCT evaluation/procedure at a study site (prospective cohort only)
- •- Received an allo-HSC infusion and to be consented in time to complete the Month 24 Visit on study (partial prospective/retrospective cohort only) or
- •- Have received their most recent allo-HSC infusion on or after January 1, 2013 (retrospective cohort only).
排除标准
- •Subjects are excluded if they meet any of the following criteria.
- •1. Previous treatment with a gene therapy product.
- •2. Receipt of an experimental transplant procedure.
研究者
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