Clinical trials: AADC Deficiency
AADC Deficiency
A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients
The overall objective of this study is to determine the safety and efficacy of AAV2-hAADC delivered to the substantia nigra pars compacta (SNc) and ventral tegmental area (VTA) in children with aromatic L-amino acid decarboxylase…
- Trial name
- AADC (NCT02852213)
- Testing
- AAV2-hAADC
- Where
- 3 sites in United States
- Volunteers
- 42 people
- Sponsor
- Krzysztof Bankiewicz
AADC Deficiency
A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
This study will have a trial phase, extension phase, and a long-term extension phase. The primary objectives of the trial phase are to assess the pharmacodynamics (PD) of eladocagene exuparvovec treatment by evaluation of…
- Trial name
- NCT04903288
- Testing
- Eladocagene Exuparvovec
- Where
- 6 sites in United States, Israel and 1 more
- Volunteers
- 13 people
- Sponsor
- PTC Therapeutics
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
An Early Clinical Trial to Evaluate VGN-R09b for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency.
This early Phase trial is to prove the safety and efficacy of VGN-R09b to treat patients with AADC deficiency.
- Trial name
- NCT05765981
- Testing
- VGN-R09b
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Shanghai Jiao Tong University School of Medicine
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC
This Phase I/II trial is to prove the efficacy and safety of AAV2-hAADC to treat patients with AADC deficiency.
- Trial name
- AADC (NCT01395641)
- Testing
- gene therapy
- Where
- Taiwan
- Volunteers
- 10 people
- Sponsor
- National Taiwan University Hospital