RecruitingNCT06224413

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

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


Sponsor

Genetix Biotherapeutics Inc.

Enrollment

120 participants

Start Date

Mar 27, 2024

Study Type

OBSERVATIONAL

Conditions

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.


Eligibility

Sex: MALE

Inclusion Criteria5

  • 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.

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Interventions

OTHERNo Intervention

This is non-interventional study.


Locations(3)

Boston Children's Hospital

Boston, Massachusetts, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

UT Southwestern Medical Center

Dallas, Texas, United States

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NCT06224413