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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
Development Status Approved
Indication Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Disease Ontology Term DOID:90123
Compound Name KEBILIDI, UPSTAZA
Compound Alias eladocagene exuparvovec
Sponsor PTC Therapeutics
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 13
Results Posted View Results

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 Low dose: 1.8E11 vg/320ul dose (approved dose)
Dose 2 High dose: 2.37E11 vg (discontinued)
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase2
Submit Date 2021-05-21
Completion Date 2028-04-30
Last Update 2025-04-10

Participation Criteria


Eligible Age 1 Year - 17 Years
Standard Ages Child
Sexes Eligible for Study ALL

Locations


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

Regulatory Information


Has US IND True
FDA Designations Accelerated Approval, Orphan Drug Designation, Priority Review, Rare Pediatric Disease Designation
Recent Updates FDA approval granted 11/13/24

Resources/Links