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

Recruiting · NCT06224413

Conditions: Cerebral Adrenoleukodystrophy (CALD)

At a glance

Research payments reported, 2025
$440,483
U.S. investigators named
2
Sites paid
2
Planned or actual enrollment
120

About the trial

Sponsor
Genetix Biotherapeutics Inc. (with Center for International Blood and Marrow Transplant Research)
Tested
No Intervention (other)
Dates
Started 2024-03-27; completion 2047-12-30
Results posted
Not yet

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 …

From ClinicalTrials.gov, last updated 2025-07-02. Full record, eligibility and contacts (opens in a new tab)

Industry funding

Genetix Biotherapeutics Inc reported $440,483 in research payments for this study in 2025 (13 payments).

Products named: Skysona

Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.

U.S. investigators named on this study

Clinicians the company named as principal investigators in its Open Payments reports.

Sites paid to run it

Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.