A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
Recruiting
Observational Study
Cerebral Adrenoleukodystrophy (CALD)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Male
- Study type
- Observational
- Participants needed
- 120 (estimated)
- Sponsor
- Genetix Biotherapeutics Inc. · Industry
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About This Trial
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 p…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
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…
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