A Postmarketing, Multicenter, Observational Study of Patients With Cerebral Adrenoleukodystrophy Treated With Elivaldogene Autotemcel (Stargazer)

What this Trial Is About

This research aims to evaluate the long-term safety outcomes of patients with Cerebral Adrenoleukodystrophy (CALD) who have been treated with elivaldogene autotemcel (eli-cel, tradename Skysona) in a real-world, post-marketing setting. The study particularly focuses on tracking newly diagnosed malignancies and assessing major functional disability-free survival over time in participants with more advanced early active CALD. This observational study includes a specific subpopulation required by the US FDA as part of accelerated approval conditions. Participants in this registry study will be followed for up to 15 years after receiving eli-cel treatment. No new investigational drug will be given during the study. The study plans to enroll 120 participants treated with eli-cel, including a subgroup of 24 participants with more advanced early active CALD, tracked separately for effectiveness outcomes. Data will be collected longitudinally to assess the safety and effectiveness of eli-cel over this extended period. During the study, participants will receive follow-up care by US-based physicians who can submit required data. Researchers will monitor adverse events including malignancies and insertional oncogenesis, as well as survival without major functional disability. Other evaluations include overall survival, serious adverse events, and detailed genetic analyses in the subpopulation with newly diagnosed hematologic malignancies. This long-term observational approach allows comprehensive safety and outcome data collection over 15 years post-infusion.

A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel