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

NCTID	NCT05394064 (View at clinicaltrials.gov)
Description	This is a Phase 1/2 randomized, blinded, dose-escalation study to evaluate the safety and efficacy of intrathecal (IT) administration of SBT101, a recombinant adeno-associated virus serotype 9 (AAV9) containing a functional copy of the human adenosine triphosphate (ATP)-binding cassette transporter subfamily D member 1 (ABCD1; hABCD1) gene, in adult patients with adrenomyeloneuropathy (AMN) aged 18-65 years. Patients will receive a single dose of SBT101 via IT route (or an imitation procedure) and will be followed for safety and efficacy for 2 years. Patients receiving SBT101 will be followed for an additional 3 years (5 total) for Safety. Patients receiving an imitation procedure will be offered the opportunity to receive SBT101 after 2 years, as data indicate. (Show More)
Development Status	Active
Indication	Adrenomyeloneuropathy (AMN)
Disease Ontology Term	DOID:10588
Compound Name	SBT101
Sponsor	SwanBio Therapeutics, Inc.
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	16
Results Posted	Not Available

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
Dose 2	3.0E14 vg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	Netherlands,United States

Has US IND	True
FDA Designations
Recent Updates	Sponsor expects to complete dosing in the Phase 1/2 PROPEL study of SBT101 early 2025 and report initial safety data from the high-dose cohort in the first half of 2025