Status:

RECRUITING

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

Lead Sponsor:

Genetix Biotherapeutics Inc.

Collaborating Sponsors:

Center for International Blood and Marrow Transplant Research

Conditions:

Cerebral Adrenoleukodystrophy (CALD)

Eligibility:

MALE

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 Sk...

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.

Key Trial Info

Start Date :

March 27 2024

Trial Type :

OBSERVATIONAL

Allocation :

ESTIMATED

End Date :

December 30 2047

Estimated Enrollment :

120 Patients enrolled

Trial Details

Trial ID

NCT06224413

Start Date

March 27 2024

End Date

December 30 2047

Last Update

July 2 2025

Active Locations (3)

Enter a location and click search to find clinical trials sorted by distance.

Page 1 of 1 (3 locations)

1

Boston Children's Hospital

Boston, Massachusetts, United States, 02115

2

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

3

UT Southwestern Medical Center

Dallas, Texas, United States, 75390

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