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Recruiting NCT06224413

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

— Observational Cerebral Adrenoleukodystrophy (CALD)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: No Intervention.
Who it may be relevant to
Registry conditions: Cerebral Adrenoleukodystrophy (CALD). Basic parameters: No limits · Male.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

  • Other No Intervention
    This is non-interventional study.

Primary outcome measures

  • Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs) [Time frame: Through 15 years postinfusion]
  • Number of Participants with Newly Diagnosed Malignancies [Time frame: Through 15 years postinfusion]
  • Number of Participants with Insertional Oncogenesis [Time frame: Through 15 years postinfusion]
  • Major Functional Disability (MFD)-Free Survival [Time frame: Through 15 years postinfusion]
Secondary outcome measures (9)
  • Overall Survival (OS) [Time frame: Through 15 years postinfusion]
  • Number of Participants with Serious Adverse Events (SAEs) [Time frame: Through 15 years postinfusion]
  • Number of Participants with Eli-cel Related AEs [Time frame: Through 15 years postinfusion]
  • Number of Participants with Presence of Insertional Oncogenesis in Subpopulation with Newly Diagnosed Hematologic Malignancy [Time frame: Through 15 years postinfusion]
  • Number of Participants with Complete Remission in Subpopulation with Newly Diagnosed Hematologic Malignancy [Time frame: Through 15 years postinfusion]
  • Relapse-free Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy [Time frame: Through 15 years postinfusion]
  • Overall Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy [Time frame: Through 15 years postinfusion]
  • 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]
  • 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]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

United States · 3 centers
  • Boston Children's Hospital — Boston
  • Children's Hospital of Philadelphia — Philadelphia
  • UT Southwestern Medical Center — Dallas

Identifiers

NCT: NCT06224413 · REG-502

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗