A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)

A Postmarketing, Prospective, Multicenter, Observational, Long-Term Safety and Effectiveness Registry Study of Patients With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)

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.

Study Overview

• Status: Recruiting
• Conditions: Cerebral Adrenoleukodystrophy (CALD)
• Intervention / Treatment: Other: No Intervention

• Study Type: Observational
• Enrollment (Estimated): 120

Contacts and Locations

• Study Contact: Name: bluebird bio; Phone Number: +1-833-999-6378; Email: clinicaltrials@bluebirdbio.com

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Recruiting
      • Boston Children's Hospital
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Children's Hospital of Philadelphia
  • Texas
    • Dallas, Texas, United States, 75390
      • Recruiting
      • UT Southwestern Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants with CALD treated with eli-cel in the post marketing setting at a center in the US that participates in the Registry Study.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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.	Other: No Intervention; This is non-interventional study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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]): 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

Secondary Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: bluebird bio
• Collaborators: Center for International Blood and Marrow Transplant Research
• Investigators: Study Director: Kiran Bhirangi, MD, bluebird bio, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): March 27, 2024
• Primary Completion (Estimated): December 30, 2047
• Study Completion (Estimated): December 30, 2047

Study Registration Dates

• First Submitted: January 16, 2024
• First Submitted That Met QC Criteria: January 16, 2024
• First Posted (Actual): January 25, 2024

Study Record Updates

• Last Update Posted (Actual): July 2, 2025
• Last Update Submitted That Met QC Criteria: June 30, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Keywords: Adrenoleukodystrophy; Adrenoleukodystrophy protein
• Additional Relevant MeSH Terms: Neurologic Manifestations; Endocrine System Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Neurobehavioral Manifestations; Demyelinating Diseases; Heredodegenerative Disorders, Nervous System; Adrenal Gland Diseases; Mental Retardation, X-Linked; Intellectual Disability; Genetic Diseases, X-Linked; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Adrenal Insufficiency; Peroxisomal Disorders; Adrenoleukodystrophy
• Other Study ID Numbers: REG-502

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No