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Clinical Trial Report

Gene Therapy Trial Report

Summary

A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants


NCTID NCT04903288 (View at clinicaltrials.gov)
Description
Indication AADC Deficiency
Compound Name KEBILIDI/UPSTAZA/eladocagene exuparvovec
Sponsor PTC Therapeutics
Funder Type Industry
Status
Active not recruiting
Enrollment Count 13

Therapy Information


Target Gene/Variant DDC
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intraparenchymal
Drug Product Type Viral vector
Target Tissue/Cell Putamen
Delivery System Viral transduction
Vector Type AAV2
Editor Type none
Dose 1 1.8E11 vg/dose, 320 ul/dose
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase2
Submit Date 2021-05-21
Completion Date 2028-04-30
Last Update 2025-01-01

Participation Criteria


Eligible Age 1 Year - 17 Years
Standard Ages Child
Eligible Sex ALL

Locations


No.of Trial Sites 6
Locations United States,Taiwan,Israel

Regulatory Information


Has US IND True
Recent Updates FDA approval granted 11/13/24

Resources/Links