Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)

NCTID	NCT05518188 (View at clinicaltrials.gov)
Description	MELPIDA is proposed for the treatment of subjects with SPG50 and targets neuronal cells to deliver a fully functional human AP4M1 cDNA copy via intrathecal injection to counter the associated neuronal loss. Outcomes will evaluate the safety and tolerability of a single dose of MELPIDA, which will be measured by the treatment-associated adverse events (AEs) and serious adverse events (SAEs). Secondarily, the trial will explore efficacy in terms of disease burden assessments. (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	Recruiting (Click here for study contact information)
Enrollment Count	4
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	1
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Phase III Study approved, begins August 2024