Observing the safety of a treatment for Cerebral Adrenoleukodystrophy
Official title: A Postmarketing, Prospective, Multicenter, Observational, Long-Term Safety and Effectiveness Registry Study of Patients With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)
Run by Genetix Biotherapeutics Inc. · NCT06224413 · Data updated 2026-11-12
This study checks how safe a treatment called elivaldogene autotemcel is for people with Cerebral Adrenoleukodystrophy over 15 years, looking at serious health issues and how well they function.
Quick facts
| Study type | Observational |
|---|---|
| Sex | Men only |
| Enrollment | 120 (estimated) |
| Sponsor | Genetix Biotherapeutics Inc. Industry-sponsored |
| Locations | 3 sites (Boston, Massachusetts and 2 other locations) |
| Status | Recruiting |
| Started | March 27, 2024 |
| Main results expected | December 30, 2047 |
| Study ends | December 30, 2047 |
| Registry record updated | July 2, 2025 |
| Trial ID | NCT06224413 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this study are patients with CALD who have been treated with eli-cel and are receiving follow-up care from a US-based physician.
✕Probably not a fit
Patients who have not been treated with eli-cel or those outside the specified geographic locations may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This study aims to assess the safety outcomes of patients with Cerebral Adrenoleukodystrophy (CALD) who have been treated with elivaldogene autotemcel (eli-cel) in a post-marketing setting. It will follow 120 participants over a period of 15 years to evaluate newly diagnosed malignancies and major functional disability-free survival. A specific cohort of 24 participants with more advanced CALD will also be monitored for effectiveness outcomes as required by the FDA. No investigational drug will be administered during this observational study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term safety and effectiveness of eli-cel for patients with CALD.
How similar studies have gone: While this study is observational and focuses on post-marketing safety, similar studies have shown promising results in assessing long-term outcomes for gene therapies.
Who can join: the full rules
Show full inclusion / exclusion 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.
Where it's running
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Boston Children's Hospital
Boston, Massachusetts, United States Recruiting -
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States Recruiting -
UT Southwestern Medical Center
Dallas, Texas, United States Recruiting
Study contacts
- Study coordinator: bluebird bio
- Email: clinicaltrials@bluebirdbio.com
- Phone: +1-833-999-6378
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.