NCT06224413

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

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

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 120 (estimated)
• Sponsor: Genetix Biotherapeutics Inc. · Industry
• Sex: Male
• Age: —
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Cerebral Adrenoleukodystrophy (CALD)

Interventions

Type	Name	Description
OTHER	No Intervention	This is non-interventional study.

Timeline

• Start date: 2024-03-27
• Primary completion: 2047-12-30
• Completion: 2047-12-30
• First posted: 2024-01-25
• Last updated: 2025-07-02

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study