Cerebral Adrenoleukodystrophy (CALD)
Conditions
Keywords
Adrenoleukodystrophy, Adrenoleukodystrophy protein
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 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.
Interventions
OTHERNo Intervention
This is non-interventional study.
Sponsors
Center for International Blood and Marrow Transplant Research
Genetix Biotherapeutics Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs) | Through 15 years postinfusion | 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 |
| 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 | Through 15 years postinfusion | 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. |
Secondary
| Measure | Time frame |
|---|---|
| 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 (OS) | 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 |
| Overall Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy | Through 15 years postinfusion |
| Number of Participants with Serious Adverse Events (SAEs) | Through 15 years postinfusion |
| Number of Participants with Eli-cel Related AEs | Through 15 years postinfusion |
| Number of Participants with Presence of Insertional Oncogenesis in Subpopulation with Newly Diagnosed Hematologic Malignancy | Through 15 years postinfusion |
Countries
United States
Contacts
Primary Contactbluebird bio
Outcome results
None listed