NCT01896102|CompletedPhase 2/3ResultsDMCFDA Drug
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A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
Sponsor study identifier
Secondary identifiers
EUDRACT_NUMBER
interventional
32
6 countries
8
Study Start
August 21, 2013
Actual (reported)Primary Completion
March 26, 2021
Actual (reported)Study Completion
March 26, 2021
Actual (reported)This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the treatment of cerebral adrenoleukodystrophy (CALD). A participant's blood stem cells were 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 were transplanted back into the participant following myeloablative conditioning. Participants in this study will be continuously followed in study LTF-304.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
March 26, 2021
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.
March 22, 2013
Actual (reported)July 11, 2013
Actual (reported)August 21, 2013
Actual (reported)March 26, 2021
Actual (reported)March 26, 2021
Actual (reported)April 25, 2022
Actual (reported)Percentage of Participants Who Were Alive and Have None of the 6 Major Functional Disabilities (MFDs) at Month 24 and Without Allo-HSCT or Rescue Cell Administration
The 6 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 was defined as: alive at 24 months post-infusion; had not developed any of the MFDs by 24 months post-infusion; had not received rescue cell administration or allo-HSCT by 24 months post-infusion; and had not withdrawn from the study or had not been lost to follow-up by 24 months post-infusion. Percentage of participants who were alive and have none of the 6 major functional disabilities (MFDs) at Month 24 were reported.
At Month 24
Proportion of Participants Who Had Experienced Either Acute ([>or=] Grade II) or Chronic Graft Versus Host Disease (GVHD) by Month 24
Acute GVHD graded on the Acute GVHD Grading Scale (I-IV): Grade I is characterized as mild disease, Grade II as moderate, Grade III as severe (involvement of any organ system), and Grade IV as life-threatening; chronic GVHD was determined by the Investigator. Percentage of participants who experienced with either acute (\>= Grade II) or chronic GVHD at Month 24 were reported.
By Month 24
Percentage of Participants Who Demonstrated Resolution of Gadolinium Positivity on Magnetic Resonance Imaging (MRI) at Month 24
At Month 24
Time to Sustained Resolution of Gadolinium Positivity on MRI
Up to Month 24
Number of Participants With Change in Total Neurologic Function Score (NFS) From Baseline up to Month 24
Baseline up to 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 Lenti-D Drug Product at a dose of greater than or equal to (\>=) 5.0 × 10\^6 CD34+ cells/kilogram (kg) (autologous CD34+ cell-enriched population that contained cells transduced with lentiviral vector encoding ABCD1 cDNA for human adrenoleukodystrophy protein, suspended in a cryopreservative solution) on Day 0.
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.
Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center
Los Angeles, California, 90095, United States
Boston Children's Hospital/Massachusetts General Hospital
Boston, Massachusetts, 02115, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Medeos SRL
Buenos Aires, C1022, Argentina
Women and Children's Hospital
North Adelaide, South Australia, 5006, Australia
Hôpital Bicêtre
Le Kremlin-Bicêtre, 94275, France
University of Leipzig
Leipzig, 04103, Germany
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.
Jakob Sieker, MD.
bluebird bio, Inc.
David Williams, MD
Boston Children's Hospital
Christine Duncan, MD
Boston Children's Hospital
Florian Eichler, MD
Massachusetts General Hospital
32 participants (actual enrollment reported by the registry).
First Submitted
March 22, 2013
First Posted
July 11, 2013
Study Start
August 21, 2013
Actual (reported)Primary Completion
Contact the study team to confirm eligibility.
Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N3JH, United Kingdom
Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, Armant M, Dansereau C, Lund TC, Miller WP, Raymond GV, Sankar R, Shah AJ, Sevin C, Gaspar HB, Gissen P, Amartino H, Bratkovic D, Smith NJC, Paker AM, Shamir E, O'Meara T, Davidson D, Aubourg P, Williams DA. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4.
Satiro de Oliveira, MD
University of California, Los Angeles
Paul Orchard, MD
University of Minnesota
Adrian Thrasher, MD, PhD
Great Ormond Street Hospital for Chidren NHS Foundation Trust
Patrick Aubourg, MD, PhD
Hôpital Bicêtre
Jorn-Sven Kuhl, MD
University of Leipzig
Nicholas Smith, MD
Women and Children's Hospital
Hernan Amartino, MD
Medeos SRL
March 26, 2021
Actual (reported)Study Completion
March 26, 2021
Actual (reported)Last Updated
April 25, 2022
Results First Posted
April 25, 2022
Record last verified: 2022-03
Bluebird bio is committed to transparency and 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. Note that given the rarity of the disease and the identifiable nature of the participants in this study, it is anticipated that the sharing of data from this study may be limited. For enquiries, please contact us at datasharing@bluebirdbio.com.
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Source: ClinicalTrials.gov.
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