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

NCT06224413 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 120

Last updated 2025-07-02

No results posted yet for this study

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)

Interventions

OTHER

No Intervention

This is non-interventional study.

Sponsors & Collaborators

Principal Investigators

  • Kiran Bhirangi, MD · bluebird bio, Inc.

Eligibility

Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-03-27
Primary Completion
2047-12-30
Completion
2047-12-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06224413 on ClinicalTrials.gov