A Postmarketing, Prospective, Multicenter, Observational, Long-Term Safety and Effectiveness Registry Study of Patients With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- bluebird bio
- 入组人数
- 120
- 试验地点
- 3
- 主要终点
- Major Functional Disability (MFD)-Free Survival
研究概览
简要总结
The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in the post-marketing setting (tradename Skysona) and to describe major functional disability (MFD)-free survival over time in participants with more advanced early active CALD. All enrolled participants with CALD treated with eli-cel in the post-marketing setting will be followed in this study for 15 years. No investigational drug product will be administered in this study. This study will enroll 120 participants with CALD treated with eli-cel in the post-marketing setting. A subpopulation of 24 participants with more advanced early active CALD will be specifically enrolled as required by the US FDA as a condition of accelerated approval and will be considered as a separate cohort for effectiveness outcomes.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Participants who fulfill the following criteria will be eligible for inclusion in this Registry Study.
- •Participant must be treated with eli-cel in the post marketing setting at a center in the United States (US) that participates in the Registry Study.
- •Participant must have provided an informed consent and/or assent to participate in Center for International Blood and Marrow Transplant Research (CIBMTR) registry.
- •Participant must have provided an informed consent and/or assent to participate in the Registry Study.
- •Participant must receive follow up care by a US-based physician with the ability to submit REG-502 data.
- •Registry Study Subpopulation inclusion:
- •Twenty-four of the 120 patients in the Registry Study must meet the following inclusion criteria which will be used to create the more advanced early active CALD subpopulation:
- •Participant must meet the above inclusion criteria and have a Loes score of 4.5 through 9.0 with GdE+ (gadolinium enhancement positivity) from an MRI performed before treatment with eli-cel and with NFS of 0 or 1 at baseline.
排除标准
- •There are no exclusion criteria for this Registry Study.
结局指标
主要结局
Major Functional Disability (MFD)-Free Survival
时间窗: Through 15 years postinfusion
MFD-free survival is defined as time from drug product infusion to either a rescue cell administration or second transplant, MFD, or death due to any cause, whichever occurs first.
Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)
时间窗: Through 15 years postinfusion
The Sponsor considers the following events to be AEIs (which should be reported as a medically significant Serious Adverse Events \[SAEs\]): * Any newly diagnosed malignancy * Neutrophil engraftment failure: defined as health care provider (HCP) decision to administer back-up cells or subsequent hematopoietic stem cell transplantation (HSCT) due to neutrophil recovery failure * Newly acquired HIV-1 or HIV-2 infection * Any newly diagnosed autoimmune disorders * Opportunistic infections * Grade 3 or higher cytopenias occurring more than 60 days after eli-cel infusion
Number of Participants with Newly Diagnosed Malignancies
时间窗: Through 15 years postinfusion
Number of Participants with Insertional Oncogenesis
时间窗: Through 15 years postinfusion
次要结局
- Overall Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy(Through 15 years postinfusion)
- Number of Participants with Clinically Significant Abnormalities on Fluorescence In-situ Hybridization (FISH) and Next Generation Sequencing (NGS) in Subpopulation with Newly Diagnosed Hematologic Malignancy(Through 15 years postinfusion)
- Number of Participants with Clinically Significant Abnormalities by Karyotyping in Bone Marrow Aspirate in Subpopulation with Newly Diagnosed Hematologic Malignancy(Through 15 years postinfusion)
- Overall Survival (OS)(Through 15 years postinfusion)
- Number of Participants with Eli-cel Related AEs(Through 15 years postinfusion)
- Number of Participants with Presence of Insertional Oncogenesis in Subpopulation with Newly Diagnosed Hematologic Malignancy(Through 15 years postinfusion)
- Number of Participants with Serious Adverse Events (SAEs)(Through 15 years postinfusion)
- Number of Participants with Complete Remission in Subpopulation with Newly Diagnosed Hematologic Malignancy(Through 15 years postinfusion)
- Relapse-free Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy(Through 15 years postinfusion)