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Clinical Trial Report

Gene Therapy Trial Report

Summary

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


NCTID NCT05518188 (View at clinicaltrials.gov)
Description
Indication Spastic Paraplegia Type 50 (SPG50)
Compound Name MELPIDA (scAAV9-AP4M1)
Sponsor Elpida Therapeutics SPC
Funder Type Industry
Status
Recruiting
Enrollment Count 4

Therapy Information


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
Dose 1 1 x 10^15 vg
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2022-08-24
Completion Date 2030-10-01
Last Update 2024-10-08

Participation Criteria


Eligible Age 4 Months - 10 Years
Standard Ages Child
Eligible Sex ALL

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


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

Resources/Links