Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
NCT ID: NCT02698579
Last Updated: 2025-02-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
64 participants
OBSERVATIONAL
2016-01-22
2038-08-31
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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CASE_ONLY
PROSPECTIVE
Study Groups
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Long-term followup
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).
No interventional drug product utilized in this follow-up study
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.
Interventions
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No interventional drug product utilized in this follow-up study
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.
Eligibility Criteria
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Inclusion Criteria
* Have received eli-cel in a parent clinical study
19 Years
MALE
No
Sponsors
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Genetix Biotherapeutics Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Vinod K Prasad, MD, FRCP
Role: STUDY_DIRECTOR
bluebird bio, Inc.
Locations
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Mattel Children's Hospital-UCLA
Los Angeles, California, United States
Lucile Packard Children's Hospital - Stanford
Palo Alto, California, United States
Boston Children's Hospital/Massachusetts General Hospital
Boston, Massachusetts, United States
University of Minnesota
Minneapolis, Minnesota, United States
Instituto Neurogenia
Caba, , Argentina
Women's and Children's Hospital
North Adelaide, , Australia
Hospital das Clínicas da Universidade de São Paulo
São Paulo, , Brazil
Hôpital Bicêtre
Le Kremlin-Bicêtre, Cedex, France
Universitätsklinikum Leipzig AöR
Leipzig, , Germany
Ospedale Pediatrico Bambino Gesù
Rome, , Italy
Prinses Maxima Center
Utrecht, , Netherlands
Royal Free London Hospital
London, England, United Kingdom
Great Ormond Street Hospital
London, , United Kingdom
Countries
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References
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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.
Related Links
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Parent Study ALD-102 protocol registrations and summary result disclosure
Parent Study ALD-104 protocol registrations and summary result disclosure
Other Identifiers
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2024-513904-33-00
Identifier Type: CTIS
Identifier Source: secondary_id
2015-002805-13
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
LTF-304
Identifier Type: -
Identifier Source: org_study_id
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