Status:
RECRUITING
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
Lead Sponsor:
Genetix Biotherapeutics Inc.
Collaborating Sponsors:
Center for International Blood and Marrow Transplant Research
Conditions:
Cerebral Adrenoleukodystrophy (CALD)
Eligibility:
MALE
Brief 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 Sk...
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 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.
Exclusion Criteria:
There are no exclusion criteria for this Registry Study.
Key Trial Info
Start Date :
March 27 2024
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 30 2047
Estimated Enrollment :
120 Patients enrolled
Trial Details
Trial ID
NCT06224413
Start Date
March 27 2024
End Date
December 30 2047
Last Update
July 2 2025
Active Locations (3)
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1
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
2
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
3
UT Southwestern Medical Center
Dallas, Texas, United States, 75390