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| Name | Class |
|---|---|
| Center for International Blood and Marrow Transplant Research | NETWORK |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All Participants | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| No Intervention | Other | This is non-interventional study. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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]):
| Through 15 years postinfusion |
| Number of Participants with Newly Diagnosed Malignancies | Through 15 years postinfusion | |
| Number of Participants with Insertional Oncogenesis | 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. | Through 15 years postinfusion |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Survival (OS) | Through 15 years postinfusion | |
| Number of Participants with Serious Adverse Events (SAEs) | Through 15 years postinfusion | |
| Number of Participants with Eli-cel Related AEs |
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Inclusion Criteria:
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:
• 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.
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Participants with CALD treated with eli-cel in the post marketing setting at a center in the US that participates in the Registry Study.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| bluebird bio | Contact | +1-833-999-6378 | clinicaltrials@bluebirdbio.com |
| Name | Affiliation | Role |
|---|---|---|
| Kiran Bhirangi, MD | bluebird bio, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Boston Children's Hospital | Recruiting | Boston | Massachusetts | 02115 | United States | |
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.
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| ID | Term |
|---|---|
| D000326 | Adrenoleukodystrophy |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| Through 15 years postinfusion |
| Number of Participants with Presence of Insertional Oncogenesis in Subpopulation with Newly Diagnosed Hematologic Malignancy | Through 15 years postinfusion |
| Number of Participants with Complete Remission in Subpopulation with Newly Diagnosed Hematologic Malignancy | Through 15 years postinfusion |
| Relapse-free Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy | Through 15 years postinfusion |
| Overall Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy | Through 15 years postinfusion |
| Number of Participants with Clinically Significant Abnormalities by Karyotyping in Bone Marrow Aspirate in Subpopulation with Newly Diagnosed Hematologic Malignancy | 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 | Through 15 years postinfusion |
| Children's Hospital of Philadelphia |
| Recruiting |
| Philadelphia |
| Pennsylvania |
| 19104 |
| United States |
| UT Southwestern Medical Center | Recruiting | Dallas | Texas | 75390 | United States |
| D009422 | Nervous System Diseases |
| D020279 | Hereditary Central Nervous System Demyelinating Diseases |
| D056784 | Leukoencephalopathies |
| D003711 | Demyelinating Diseases |
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D008661 | Metabolism, Inborn Errors |
| D018901 | Peroxisomal Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D000309 | Adrenal Insufficiency |
| D000307 | Adrenal Gland Diseases |
| D004700 | Endocrine System Diseases |