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
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- bluebird bio
- 入组人数
- 59
- 试验地点
- 13
- 主要终点
- Incidence and timing of neutrophil engraftment.
研究概览
简要总结
Study ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- — 至 17 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Provide informed consent from a competent custodial parent 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 institution's IRB/IEC and all other local requirements.
- •Be male and ≤17 years of age at the time of treatment, for retrospective and partial prospective/retrospective subjects, or at the time of parental/guardian consent and, where appropriate, subject assent, for prospective subjects.
- •Have a confirmed diagnosis of CALD as defined by abnormal VLCFA profile and cerebral lesion on brain MRI.
- •Depending on the cohort, the subject must:
- •Be scheduled for allo-HSCT evaluation at a study site (prospective cohort only),
- •Have received an allo-HSC infusion and 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
- •Previous treatment with a gene therapy product.
- •Receipt of an experimental transplantation procedure.
结局指标
主要结局
Incidence and timing of neutrophil engraftment.
时间窗: 1-48 (± 1) months post allo-HSC infusion
Frequency and severity of Criteria for Adverse Events (CTCAE) ≥Grade 3 AEs, CTCAE ≥Grade 3 infections, and all SAEs.
时间窗: 1-48 (± 1) months post allo-HSC infusion
Incidence of ≥Grade II acute GVHD.
时间窗: 1-48 (± 1) months post allo-HSC infusion
Incidence of chronic GVHD.
时间窗: 1-48 (± 1) months post allo-HSC infusion
Incidence of transplant-related mortality (TRM).
时间窗: Through 100 and 365 days post allo-HSC infusion
TRM is defined as death due to any transplantation-related cause other than disease progression.
Incidence and timing of platelet engraftment
时间窗: 1-48 (± 1) months post allo-HSC infusion
Incidence of primary donor-derived chimerism of ≥50%.
时间窗: by 100 days post allo-HSC infusion
Number and duration of intensive care unit stay.
时间窗: 1-48 (± 1) months post allo-HSC infusion
Incidence of engraftment failure or allograft rejection.
时间窗: 1-48 (± 1) months post allo-HSC infusion
Proportion of subjects who experience either ≥Grade II acute (Graft versus Host Disease) GVHD or chronic GVHD.
时间窗: 1-48 (± 1) months post allo-HSC infusion
Number of emergency room visits.
时间窗: 1-48 (± 1) months post allo-HSC infusion
Number and duration of in-patient hospitalization.
时间窗: 1-48 (± 1) months post allo-HSC infusion
次要结局
- MFD-free survival(48 (± 2) months post allo-HSC infusion)
- Change from Baseline in Loes score(1-48 (± 2) months post allo-HSC infusion)
- Change from Baseline in Neurological Function Score (NFS)(1-48 (± 2) months post allo-HSC infusion)
- Frequency and timing of resolution of gadolinium enhancement on MRI, if applicable(1-48 (± 2) months post allo-HSC infusion)
- Incidence of Major Functional Disabilities (MFDs).(1-48 (± 2) months post allo-HSC infusion)
- Overall survival(48 (± 2) months post allo-HSC infusion)