Phase 3, Open-label Study to Assess the Efficacy and Safety of a Single Lumbar Intrathecal Administration of MELPIDA in Individuals with Hereditary Spastic Paraplegia Type 50 (SPG50) Versus Matched Prospective Concurrent Controls.

NCTID	NCT06692712 (View at clinicaltrials.gov)
Description	Phase 3, open-label study to assess the efficacy and safety of a single lumbar intrathecal administration of MELPIDA in individuals with Hereditary Spastic Paraplegia Type 50 (SPG50). (Show More)
Development Status	Active
Indication	Hereditary Spastic Paraplegia Type 50
Disease Ontology Term	DOID:0110802
Compound Name	MELPIDA
Compound Description	scAAV9-AP4M1
Sponsor	Elpida Therapeutics SPC
Funder Type	Industry
Recruitment Status	Not yet recruiting
Enrollment Count	24
Results Posted	Not Available

Target Gene/Variant	AP4M1
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	1E15 vg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites
Locations