A Follow-Up Study to Evaluate the Safety and Clinical Outcomes of Patients With Non-Malignant Disease Who Have Undergone Hematopoietic Stem Cell Transplantation With MGTA-456
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Incidence of related adverse events
研究概览
简要总结
A follow-up study to evaluate the safety and clinical outcomes of patients with inherited metabolic disorders (IMD) who have undergone hematopoietic stem cell transplantation (HSCT) with MGTA-456
详细描述
This is a follow-up study to evaluate the long-term safety and efficacy outcomes of patients with inherited metabolic disorders (IMDs) who received MGTA-456 for HSCT in the core study. MGTA-456 is an expanded CD34+ cell therapy product candidate given after myeloablative conditioning to induce rapid and sustained hematopoietic engraftment. In patients with selected IMDs, transplant is expected to replace defective or missing protein, and preserve neurodevelopment. Patients with Hurler syndrome (also referred to as mucopolysaccharidosis-1H (MPS-1H)), cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD) enrolled in the core study will be eligible to participate in this follow-up evaluation.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •An Institutional Review Board (IRB)/Independent Ethics Committee-approved written informed consent form must be signed and dated by the patient or legal guardian. Study assents will also be prepared for children and adolescents to review when applicable.
- •Patient completed an IMD study in the MGTA-456 program and was administered MGTA-456 for HSCT.
排除标准
- •Patients enrolled in an IMD study in the MGTA-456 program who did not receive MGTA-456 or were withdrawn from the core study.
结局指标
主要结局
Incidence of related adverse events
时间窗: 2 years
Incidence of chronic graft versus host disease
时间窗: 2 years
Event-free survival
时间窗: 2 years
Very long chain fatty acid blood level (ug/mL) in cALD patients
时间窗: 2 years
Incidence of late hematological graft failure
时间窗: 2 years
Proportion of subjects without gadolinium enhancement on MRI over time
时间窗: 2 years
Alpha-iduronidase blood enzyme level (nmol/hr/mg) in Hurler patients
时间窗: 2 years
Incidence of serious adverse events
时间窗: 2 years
Overall survival
时间窗: 2 years
Change in cALD Neurologic Function Score over time
时间窗: 2 years
次要结局
未报告次要终点