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

PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

13

Participants

Timeline

Start Date

May 12, 2021

Primary Completion Date

May 22, 2023

Study Completion Date

April 30, 2028

Conditions
AADC Deficiency
Interventions
GENETIC

Eladocagene Exuparvovec

Four 0.08 milliliters (mL) infusions at a dose of 0.45×10\^11 vg and a volume of 80 microliters (μl) per site to 4 sites (2 per putamen), for the total dose of 1.8×10\^11 vg and a total volume of 320 μl per participant.

Trial Locations (6)

10041

National Taiwan University Hospital, Department of Pediatrics and Medical Genetics, Taipei

27705

Duke University Hospital, Durham

45229

Cincinnati Children's Hospital Medical Center, Cincinnati

77030

Texas Children's Hospital, Houston

5262000

Chaim Sheba Medical Center, Ramat Gan

02115

Boston Children's Hospital, Boston

Sponsors

Lead Sponsor

All Listed Sponsors
lead

PTC Therapeutics

INDUSTRY

NCT04903288 - A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants | Biotech Hunter | Biotech Hunter