Sponsor study identifier
Secondary identifiers
observational
64
9 countries
13
Study Start
January 22, 2016
Actual (reported)Primary Completion
August 2038
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
August 2038
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
August 2038
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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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 22, 2016
Actual (reported)February 17, 2016
Actual (reported)March 3, 2016
Actual (reported)August 2038
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
August 2038
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Major functional disability (MFD)-free survival
The MFDs are loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, complete loss of voluntary movement.
15 years post-drug-product infusion
Number of participants with malignancies
15 years post-drug-product infusion
Number of participants who experience graft versus host disease (GVHD)
15 years post-drug-product infusion
Participants who have received Lenti-D Drug Product in a parent clinical study (bluebird bio-sponsored clinical studies ALD-102 and ALD-104) and who meet the eligibility criteria for the Study LTF-304 will be followed in this long-term followup study for 13 years (for a total of 15 years of follow-up after drug product infusion in the parent studies).
Participants received a single IV infusion of Lenti-D Drug Product (also known as elivaldogene autotemcel or eli-cel) in either parent Study ALD-102 or ALD-104. The objectives of this long-term follow-up study are to assess long-term safety and efficacy following completion of participation in parent studies. Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant are conducted in this study.
Participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in a parent clinical study will be expected to participate in this long-term follow-up study.
Mattel Children's Hospital-UCLA
Los Angeles, California, 90095, United States
Lucile Packard Children's Hospital - Stanford
Palo Alto, California, 94304, United States
Boston Children's Hospital/Massachusetts General Hospital
Boston, Massachusetts, 02115, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Instituto Neurogenia
Caba, Argentina
Women's and Children's Hospital
North Adelaide, Australia
Hospital das Clínicas da Universidade de São Paulo
Eichler F, Duncan CN, Musolino PL, Lund TC, Gupta AO, De Oliveira S, Thrasher AJ, Aubourg P, Kuhl JS, Loes DJ, Amartino H, Smith N, Folloni Fernandes J, Sevin C, Sankar R, Hussain SA, Gissen P, Dalle JH, Platzbecker U, Downey GF, McNeil E, Demopoulos L, Dietz AC, Thakar HL, Orchard PJ, Williams DA. Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2024 Oct 10;391(14):1302-1312. doi: 10.1056/NEJMoa2400442.
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.
Vinod K Prasad, MD, FRCP
Genetix Biotherapeutics Inc.
64 participants (actual enrollment reported by the registry).
First Submitted
February 17, 2016
First Posted
March 3, 2016
Study Start
January 22, 2016
Actual (reported)Number of participants with immune-related adverse events (AEs)
15 years post-drug-product infusion
Number of participants with new or worsening hematologic disorders
15 years post-drug-product infusion
Number of participants with new or worsening neurologic disorders
15 years post-drug-product infusion
Number of participants who undergo subsequent stem cell transplantation
15 years post-drug-product infusion
Change from baseline in neurological function score (NFS)
15 years post-drug-product infusion
Number of participants without gadolinium enhancement (GdE) status on magnetic resonance imaging (MRI)
15 years post-drug-product infusion
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Contact the study team to confirm eligibility.
São Paulo, 05403-000, Brazil
Hôpital Bicêtre
Le Kremlin-Bicêtre, Cedex, 94275, France
Universitätsklinikum Leipzig AöR
Leipzig, 04103, Germany
Ospedale Pediatrico Bambino Gesù
Rome, 00165, Italy
Prinses Maxima Center
Utrecht, 3584, Netherlands
Royal Free London Hospital
London, England, NW3 2QG, United Kingdom
Great Ormond Street Hospital
London, England, United Kingdom
Primary Completion
August 2038
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
August 2038
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Last Updated
September 14, 2026
Record last verified: 2026-09
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 to maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.
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Source: ClinicalTrials.gov.
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