NCT03852498|CompletedPhase 3ResultsDMCFDA Drug
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A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects ≤17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)
Sponsor study identifier
Secondary identifiers
EUDRACT_NUMBER
NIH
interventional
35
6 countries
8
Study Start
January 24, 2019
Actual (reported)Primary Completion
July 24, 2023
Actual (reported)Study Completion
July 24, 2023
Actual (reported)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 expected, although participants follow-up remains ongoing in the long-term follow-up Study LTF-304 (NCT02698579).
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
July 24, 2023
Actual (reported)Click on a node to explore related trials.
Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
January 24, 2019
Actual (reported)February 13, 2019
Actual (reported)February 25, 2019
Actual (reported)July 24, 2023
Actual (reported)July 24, 2023
Actual (reported)May 24, 2024
Actual (reported)Percentage of Participants Who Were Alive and Have None of the 6 Major Functional Disabilities (MFDs) at Month 24
The MFDs consisted of loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, complete loss of voluntary movement. Month-24 MFD-Free survival criteria defined as: alive at 24 months post infusion; have not developed any of the MFDs by 24 months post infusion; have not received rescue cell administration or allo-hematopoietic stem cell transplantation (HSCT) by 24 months post infusion; and have not withdrawn from the study or have not been lost to follow-up by 24 months post infusion.
At Month 24
Percentage of Participants Who Achieved Neutrophil Engraftment After Drug Product Infusion
Neutrophil engraftment is defined as achieving 3 consecutive absolute neutrophil count (ANC) laboratory values of \>= 0.5x10\^9 cells/liter (L) (after initial post-infusion nadir) obtained on different days by 42 days postinfusion of eli-cel (Rel Day 43).
By 42 days post-drug infusion
Percentage of Participants Without Gadolinium Enhancement (i.e. GdE-) on Magnetic Resonance Imaging (MRI) at Month 24
At Month 24
Number of Participants With Change in Neurologic Function Score (NFS) From Baseline to Month 24
Baseline up to Month 24
Number of Participants Who Achieved Stable NFS at Month 24
At Month 24
Major Functional Disability (MFD)-Free Survival Rate
At 24 months after Lenti-D drug infusion
Overall Survival Rate
At 24 months after Lenti-D drug infusion
Participants received a single intravenous (IV) infusion of eli-cel (also referred to as Lenti-D Drug Product) on Day 1 at a dose of \> or = 5.0\*10\^6 CD34+ cells/kilogram (kg). All participants received myeloablative conditioning with busulfan and fludarabine over a number of days prior to drug product infusion.
Participants received a single IV infusion of Lenti-D Drug Product.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Lucile Packard Children's Hospital
Palo Alto, California, 94304, United States
Boston Children's Hospital/Massachusetts General Hospital
Boston, Massachusetts, 02215, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Hôpital Robert Debré
Paris, 75019, France
Universitätsklinikum Leipzig AöR
Leipzig, 04103, Germany
Ospedale Pediatrico Bambino Gesù
Rome, 00165, Italy
Prinses Maxima Center
Utrecht, 3508AB, Netherlands
Duncan CN, Bledsoe JR, Grzywacz B, Beckman A, Bonner M, Eichler FS, Kuhl JS, Harris MH, Slauson S, Colvin RA, Prasad VK, Downey GF, Pierciey FJ, Kinney MA, Foos M, Lodaya A, Floro N, Parsons G, Dietz AC, Gupta AO, Orchard PJ, Thakar HL, Williams DA. Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2024 Oct 10;391(14):1287-1301. doi: 10.1056/NEJMoa2405541.
Himal Lal Thakar, MD
bluebird bio, Inc.
35 participants (actual enrollment reported by the registry).
First Submitted
February 13, 2019
First Posted
February 25, 2019
Study Start
January 24, 2019
Actual (reported)Contact the study team to confirm eligibility.
UCL-ICH/Great Ormond Street Hospital
London, WC1N3JH, United Kingdom
Primary Completion
July 24, 2023
Actual (reported)Study Completion
July 24, 2023
Actual (reported)Last Updated
May 24, 2024
Results First Posted
May 24, 2024
Record last verified: 2024-04
Bluebird bio is committed to transparency. Appropriately de-identified patient-level datasets and supporting documents may be shared following attainment of applicable marketing approvals associated with this study and consistent with criteria established by bluebird bio and/or industry best practices in order to maintain the privacy of study participants. For enquiries, please contact bluebird bio at datasharing@bluebirdbio.com.
Download the complete ClinicalTrials.gov record imported by Syfrah, including fields not shown in this page. The registry may contain a newer revision.
Source: ClinicalTrials.gov.
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