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

NCT06224413 | Recruiting FDA Drug

• 1 other identifier: REG-502
• Study Type: observational
• Target: 120
• Locations: 1 country
• Sites: 3

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.

Conditions

Cerebral Adrenoleukodystrophy (CALD)

Keywords

Adrenoleukodystrophy; Adrenoleukodystrophy protein

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (9)

• 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

Study Arms (1)

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

Interventions

No Intervention OTHER

This is non-interventional study.

All Participants

Eligibility Criteria

• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

Sponsors & Collaborators

• Genetix Biotherapeutics Inc.: lead
• Center for International Blood and Marrow Transplant Research: collaborator

Study Sites (3)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

MeSH Terms

Conditions

Adrenoleukodystrophy

Condition Hierarchy (Ancestors)

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

Study Officials

• Kiran Bhirangi, MD

  bluebird bio, Inc.

  STUDY DIRECTOR

Central Study Contacts

bluebird bio

CONTACT

+1-833-999-6378; clinicaltrials@bluebirdbio.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 15 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 16, 2024
• First Posted: January 25, 2024
• Study Start: March 27, 2024
• Primary Completion (Estimated): December 30, 2047
• Study Completion (Estimated): December 30, 2047
• Last Updated: July 2, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will share

Locations

US (3)