13
Participants
Start Date
May 12, 2021
Primary Completion Date
May 22, 2023
Study Completion Date
April 30, 2028
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.
National Taiwan University Hospital, Department of Pediatrics and Medical Genetics, Taipei
Duke University Hospital, Durham
Cincinnati Children's Hospital Medical Center, Cincinnati
Texas Children's Hospital, Houston
Chaim Sheba Medical Center, Ramat Gan
Boston Children's Hospital, Boston
Lead Sponsor
PTC Therapeutics
INDUSTRY