Actively Recruiting
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
Led by Genetix Biotherapeutics Inc. · Updated on 2025-07-02
120
Participants Needed
3
Research Sites
1239 weeks
Total Duration
On this page
Sponsors
G
Genetix Biotherapeutics Inc.
Lead Sponsor
C
Center for International Blood and Marrow Transplant Research
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant must be treated with eli-cel in the post marketing setting at a center in the United States 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 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.
- For the subpopulation: Participant must have a Loes score of 4.5 through 9.0 with gadolinium enhancement positivity from an MRI before eli-cel treatment and a neurologic function score of 0 or 1 at baseline.
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
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Trial Site Locations
Total: 3 locations
1
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
2
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
3
UT Southwestern Medical Center
Dallas, Texas, United States, 75390
Actively Recruiting
Research Team
B
bluebird bio
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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