An observational study in Cerebral Adrenoleukodystrophy (CALD), sponsored by Genetix Biotherapeutics Inc.. Recruiting at 3 sites in United States. Open to male participants. Per ClinicalTrials.gov, last updated 2025-07-02.
Sponsored by Genetix Biotherapeutics Inc. · Observational
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.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
This study's planned enrollment of 120 is close to the median of 120 across 19 observational studies indexed under Adrenoleukodystrophy.
Browse Adrenoleukodystrophy studies →Genetix Biotherapeutics Inc. is the lead sponsor of 2 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Participants with CALD treated with eli-cel in the post marketing setting at a center in the US that participates in the Registry Study.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Participants who fulfill the following criteria will be eligible for inclusion in this Registry Study.
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:
Exclusion Criteria:
There are no exclusion criteria for this Registry Study.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants with CALD treated with eli-cel in the post marketing setting will be followed in this registry study for up to 15 years after eli-cel infusion to collect real-world longitudinal data, and evaluate the outcomes.
Other: No Intervention
This is non-interventional study.
Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)
The Sponsor considers the following events to be AEIs (which should be reported as a medically significant Serious Adverse Events \[SAEs\]): * Any newly diagnosed malignancy * Neutrophil engraftment failure: defined as health care provider (HCP) decision to administer back-up cells or subsequent hematopoietic stem cell transplantation (HSCT) due to neutrophil recovery failure * Newly acquired HIV-1 or HIV-2 infection * Any newly diagnosed autoimmune disorders * Opportunistic infections * Grade 3 or higher cytopenias occurring more than 60 days after eli-cel infusion
Time frame: Through 15 years postinfusion
Number of Participants with Newly Diagnosed Malignancies
Time frame: Through 15 years postinfusion
Number of Participants with Insertional Oncogenesis
Time frame: Through 15 years postinfusion
Major Functional Disability (MFD)-Free Survival
MFD-free survival is defined as time from drug product infusion to either a rescue cell administration or second transplant, MFD, or death due to any cause, whichever occurs first.
Time frame: Through 15 years postinfusion
Overall Survival (OS)
Time frame: Through 15 years postinfusion
Number of Participants with Serious Adverse Events (SAEs)
Time frame: Through 15 years postinfusion
Number of Participants with Eli-cel Related AEs
Time frame: Through 15 years postinfusion
Number of Participants with Presence of Insertional Oncogenesis in Subpopulation with Newly Diagnosed Hematologic Malignancy
Time frame: Through 15 years postinfusion
Number of Participants with Complete Remission in Subpopulation with Newly Diagnosed Hematologic Malignancy
Time frame: Through 15 years postinfusion
Relapse-free Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy
Time frame: Through 15 years postinfusion
Overall Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy
Time frame: Through 15 years postinfusion
Number of Participants with Clinically Significant Abnormalities by Karyotyping in Bone Marrow Aspirate in Subpopulation with Newly Diagnosed Hematologic Malignancy
Time frame: Through 15 years postinfusion
Number of Participants with Clinically Significant Abnormalities on Fluorescence In-situ Hybridization (FISH) and Next Generation Sequencing (NGS) in Subpopulation with Newly Diagnosed Hematologic Malignancy
Time frame: Through 15 years postinfusion
Plan to share: Yes — Bluebird bio is committed to transparency and appropriately de-identified patient-level datasets and supporting documents may be shared (if contractually or otherwise legally permitted ) following completion of this study, submission of all applicable regulatory submissions and consistent with criteria established by bluebird bio, our collaborators (CIBMTR) and/or industry best practices to protect confidential information and maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.
No publications or documents are linked to this record.
Started Mar 2024 (actual). Primary completion is estimated for Dec 2047, 285 months after the start.
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Genetix Biotherapeutics Inc.