A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
Completed · Phase 3 · NCT03852498
Conditions: Cerebral Adrenoleukodystrophy (CALD)
At a glance
- Research payments reported, 2025
- $23,527
- U.S. investigators named
- 1
- Sites paid
- 1
- Planned or actual enrollment
- 35
About the trial
- Sponsor
- Genetix Biotherapeutics Inc.
- Tested
- Lenti-D (genetic)
- Dates
- Started 2019-01-24; completion 2023-07-24
- Results posted
- Yes
The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and fludarabine in participants with CALD. A participant's blood stem cells will be collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells will be transplanted back into the participant following myeloablative conditioning. Enrollment and treatment in Study ALD-104 have been completed and further enrollment in this study is not expe…
From ClinicalTrials.gov, last updated 2024-05-24. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Genetix Biotherapeutics Inc reported $23,527 in research payments for this study in 2025 (2 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
- Regents of the University of Minnesota $23,527
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.