A Postmarketing, Prospective, Multicenter, Observational, Long-Term Safety and Effectiveness Registry Study of Patients With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)
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.
⁃ 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.