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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
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