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Clinical Trial Report

Gene Therapy Trial Report

Summary

A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)


NCTID NCT05394064 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Adrenomyeloneuropathy (AMN)
Disease Ontology Term DOID:10588
Compound Name SBT101
Sponsor SwanBio Therapeutics, Inc.
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 8 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant ABCD1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intrathecal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 1.0E14 vg (n=4)
Dose 2 3.0E14 vg (n=4)
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2022-05-16
Completion Date 2025-08-31
Last Update 2025-10-31

Participation Criteria


Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE

Locations


No.of Trial Sites 2
Locations Netherlands,United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Study was terminated for business/strategic reasons, not due to safety concerns

Resources/Links