• In The United States

Active, recruiting:

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

Learn More https://clinicaltrials.gov/ct2/show/NCT04903288?cond=aadc&draw=2&rank=4

○ Current Active sites in the United States:

- United States, Texas

• Texas Children's Hospital (Recruiting) - 
• Contact: Sarah Wisor (sxwisor@texaschildrens.org)

- United States, California

• Lucile Packard Children's Hospital Stanford (Not Yet Recruiting) - 
• Contact: Matthew Sklar (msklar7@stanford.edu)
• Anthony Bet (abet0915@stanford.edu) 

- United States, Massachusetts

• Boston Children's Hospital (Not Yet Recruiting) - 
• Contact: Adam Porter (adam.porter@childrens.harvard.edu)

- United States, North Carolina

• Duke University Medical Center (Not Yet Recruiting) - 
• Contact: Claudia Pamanes (Claudia.pamanes@duke.edu)

- United States, Ohio

• Cincinnati Children's Hospital Medical Center (Not Yet Recruiting)

● A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients (AADC)

Learn More  https://clinicaltrials.gov/ct2/show/NCT02852213?cond=aadc&draw=2&rank=1 

○ Current Active sites in the United States:

                 - United States, California

                       ○ University of California, San Francisco (Active, Not Recruiting)

                  - United States, Ohio

                       ○ Nationwide Children's Hospital, Columbus (Recruiting)

• Contact: Shweta Saraswat (shweta.saraswat@nationwidechildrens.org)
• Principal Investigator: Jeffery Leonard, MD
• The Ohio State Medical Center, Columbus
• Contact: Andrea Davis (andrea.hesse@osumc.edu, 614-688-6412)
• Principal Investigators: Russell Lonser, MD and Krystof Bankiewick
  

In Japan/Taiwan

Past trials:

Target area of the brain: Putamen

● National Taiwan University Hospital

A study published in 2017 details an open-label phase ½ trial that was performed at the National Taiwan University Hospital in Taipei, Taiwan in 2014-2015. The findings of this study suggest that intraputaminal injection of the AAV2-hAADC is well tolerated, and might improve motor development in children with AADC deficiency.

Learn More Here: https://www.thelancet.com/action/showPdf?pii=S2352-4642%2817%2930125-6

● Jichi Medical University

This study was planned as an open-label, phase 1/2 trial at Jichi Medical University Hospital (Tochigi, Japan) to investigate the effects of gene therapy in patients with AADC deficiency.

All six of the patients in our study showed marked improvement in their motor function after gene transfer. Although five patients with the severe phenotype had been bedridden before treatment, one patient (Patient 2) became able to walk using a walker, and two patients (Patients 1 and 5) were able to stand with support.

Learn More Here:

https://academic.oup.com/brain/article/142/2/322/5296575

In Poland

Past trials:

● Bródno Mazovia Hospital, Warsaw, Poland

Target area of the brain: Substantia Nigra Pars Compacta & Ventral Tegmental

The sister facility to the one located at the University of California in San Francisco, Professor Krystof Bankiewicz’s team in Poland has been seeing success with the same clinical trial method used for gene therapy as the United States.

So far, a total of 13 individuals suffering with AADC have received the pioneering and potentially life saving brain surgery in Europe.

Learn More Here:

https://journals.viamedica.pl/neurologia_neurochirurgia_polska/article/view/PJNNS.a2020.0046/51160