Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
Active, not recruiting · NCT02698579
Conditions: Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
At a glance
- Research payments reported, 2025
- $778,026
- U.S. investigators named
- 6
- Sites paid
- 6
- Planned or actual enrollment
- 64
About the trial
- Sponsor
- Genetix Biotherapeutics Inc.
- Tested
- No interventional drug product utilized in this follow-up study (genetic)
- Dates
- Started 2016-01-22; completion 2038-08
- Results posted
- Not yet
This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical study (Study ALD-102 or Study ALD-104). After completing a parent clinical study (approximately 2 years), eligible participants will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in this study.
From ClinicalTrials.gov, last updated 2026-09-14. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Genetix Biotherapeutics Inc reported $778,026 in research payments for this study in 2025 (44 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
- Satiro N. De Oliveira, M.D. Pediatric Hematology & Oncology · Los Angeles, CA
- Ami J. Shah, MD Pediatric Hematology & Oncology · Palo Alto, CA
- Christine N. Duncan, M.D. Pediatric Hematology & Oncology · Boston, MA
- Florian S. Eichler, MD Neurology · Boston, MA
- David A. Williams, MD Pediatric Hematology & Oncology · Boston, MA
- Paul J. Orchard, MD Pediatric Hematology & Oncology · Minneapolis, MN
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Regents of the University of Minnesota $346,735
- Board of Trustees of the Leland Stanford Jr University $140,484
- Ronald Reagan UCLA Medical Center, CA $88,359
- Massachusetts General Hospital, MA $81,556
- Boston Children's Hospital, MA $61,169
- Brigham and Women's Hospital, MA $59,722
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.