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

Recruiting

• Conditions: Cerebral Adrenoleukodystrophy (CALD)
• Interventions: Other: No Intervention

• Registration Number: NCT06224413
• Lead Sponsor: bluebird bio

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-01-16
• Study First Submitted QC: 2024-01-16
• Study First Posted: 2024-01-25
• Study Start: 2024-03-27
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-09
• Last Update Posted: 2024-04-10
• Primary Completion: 2047-12-30
• Study Completion: 2047-12-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Male
• Target Recruitment: 120

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.

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Exclusion Criteria

There are no exclusion criteria for this Registry Study.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
All Participants	No Intervention	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.

Primary Outcome Measures

Name	Time	Method
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.
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

Secondary Outcome Measures

Name	Time	Method
Overall Survival 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
Number of Participants with Clinically Significant Abnormalities by Karyotyping in Bone Marrow Aspirate in Subpopulation with Newly Diagnosed Hematologic Malignancy	Through 15 years postinfusion
Overall Survival (OS)	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 Serious Adverse Events (SAEs)	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

Trial Locations

Locations (2)

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States