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.
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.
None
Participants with CALD treated with eli-cel in the post marketing setting at a center in the US that participates in the Registry Study.
Cohort
Prospective
No Intervention
Other interventionThis is non-interventional study.
Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)
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
Number of Participants with Insertional Oncogenesis
Major Functional Disability (MFD)-Free Survival
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.
Overall Survival (OS)
Number of Participants with Serious Adverse Events (SAEs)
Number of Participants with Eli-cel Related AEs
Number of Participants with Presence of Insertional Oncogenesis in Subpopulation with Newly Diagnosed Hematologic Malignancy
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Genetix Biotherapeutics Inc.
Lead sponsor
Center for International Blood and Marrow Transplant Research
Collaborator
