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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
NCTID
NCT03852498
(View at clinicaltrials.gov)
Description
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).
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Development Status
Approved
Indication
Cerebral Adrenoleukodystrophy (CALD)
Disease Ontology Term
DOID:10588
Compound Name
SKYSONA
Compound Alias
elivaldogene autotemcel
Sponsor
bluebird bio
Funder Type
Industry
Recruitment Status
Completed
Enrollment Count
35
Results Posted
View Results
Therapy Information
Target Gene/Variant
ABCD1
Therapy Type
Gene transfer
Therapy Route
Ex-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intravenous
Drug Product Type
Autologous cells
Target Tissue/Cell
CD34+ cells
Delivery System
Viral transduction
Vector Type
LV
Editor Type
none
Dose 1
At least 5.0E6 CD34+ cells/kg; MOI = 40
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase3
Submit Date
2019-02-13
Completion Date
2023-07-24
Last Update
2024-05-24
Participation Criteria
Eligible Age
<=17 Years
Standard Ages
Child
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
8
Locations
Netherlands,United States,Italy,United Kingdom,France,Germany
Regulatory Information
Has US IND
True
FDA Designations
Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates
FDA approval granted 9/16/22; Cost/treatment $3M
Resources/Links
Clinical Publications
Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
Protocol
FDA Approval Documents
Statistical Analysis Plan
Clinical Trial Protocol
Related NCTID
Phase 2/3: NCT01896102
Long Term Follow-Up: NCT02698579