Stargazer
A Postmarketing, Prospective, Multicenter, Observational, Long-Term Safety and Effectiveness Registry Study of Patients With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)
Sponsor study identifier
observational
120
1 country
3
Study Start
March 27, 2024
Actual (reported)Primary Completion
December 30, 2047
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
December 30, 2047
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
December 30, 2047
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
January 16, 2024
Actual (reported)January 25, 2024
Actual (reported)March 27, 2024
Actual (reported)December 30, 2047
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
December 30, 2047
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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
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
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
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
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.
This is non-interventional study.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Participants with CALD treated with eli-cel in the post marketing setting at a center in the US that participates in the Registry Study.
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
UT Southwestern Medical Center
Dallas, Texas, 75390, United States
Kiran Bhirangi, MD
bluebird bio, Inc.
First Submitted
January 16, 2024
First Posted
January 25, 2024
Study Start
March 27, 2024
Actual (reported)Primary Completion
December 30, 2047
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Contact the study team to confirm eligibility.
Study Completion
December 30, 2047
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Last Updated
July 2, 2025
Record last verified: 2025-06
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.
Download the complete ClinicalTrials.gov record imported by Syfrah, including fields not shown in this page. The registry may contain a newer revision.
Source: ClinicalTrials.gov.
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