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NCT Number: NCT06224413

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

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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Key information

Conditions

Cerebral Adrenoleukodystrophy (cALD) Adrenal Gland Diseases Adrenal Insufficiency Adrenoleukodystrophy Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Demyelinating Diseases Endocrine System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Hereditary Central Nervous System Demyelinating Diseases Heredodegenerative Disorders, Nervous System Intellectual Disability Leukoencephalopathies Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutritional and Metabolic Diseases Peroxisomal Disorders X-Linked Intellectual Disability

Sex eligibility

Male

Study type

Observational

Primary location

Boston Children's Hospital, Boston, Massachusetts, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

No intervention

Other

This is non-interventional study.

Primary outcomes

  1. Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)

    Time frame: 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

  2. Number of Participants with Newly Diagnosed Malignancies

    Time frame: Through 15 years postinfusion

  3. Number of Participants with Insertional Oncogenesis

    Time frame: Through 15 years postinfusion

  4. Major Functional Disability (MFD)-Free Survival

    Time frame: 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 outcomes

  1. Overall Survival (OS)

    Time frame: Through 15 years postinfusion

  2. Number of Participants with Serious Adverse Events (SAEs)

    Time frame: Through 15 years postinfusion

  3. Number of Participants with Eli-cel Related AEs

    Time frame: Through 15 years postinfusion

  4. Number of Participants with Presence of Insertional Oncogenesis in Subpopulation with Newly Diagnosed Hematologic Malignancy

    Time frame: Through 15 years postinfusion

  5. Number of Participants with Complete Remission in Subpopulation with Newly Diagnosed Hematologic Malignancy

    Time frame: Through 15 years postinfusion

  6. Relapse-free Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy

    Time frame: Through 15 years postinfusion

  7. Overall Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy

    Time frame: Through 15 years postinfusion

  8. 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

  9. 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

Study contacts

Contact information is provided by the study sponsor or research team.

bluebird bio

CONTACT

[email protected]

+1-833-999-6378

Sponsors and collaborators

Lead sponsor

Genetix Biotherapeutics Inc.

Industry

Collaborators

  • Center for International Blood and Marrow Transplant Research

Registry information

Official study title

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

Acronym: Stargazer

Important dates

Study start
2024
Primary completion
2047
Study completion
2047
First posted
Jan 25, 2024
Registry last updated
Jul 2, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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