Clinical trials: AADC Deficiency, active
Aromatic L-amino Acid Decarboxylase Deficiency
A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency
This trial includes dose-escalating part (phase 1) and dose confirming part, to prove the safety and efficacy of VGN-R09b to treat patients with severe AADC deficiency
- Trial name
- NCT06432140
- Testing
- VGN-R09b injection
- Where
- China
- Volunteers
- 13 people
- Sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.
Neurology, Aromatic Amino Acid Decarboxylase Deficiency
Newborn Screening and Early Diagnosis of AADC Deficiency in the Ligurian Population
Aromatic L-amino acid decarboxylase deficiency (AADC-D) is a rare genetic disorder that affects the production of neurotransmitters in the brain and can cause severe neurological and developmental problems.
- Conditions
- Neurology, Aromatic Amino Acid Decarboxylase Deficiency, Pediatric Neurological Disorder
- Trial name
- NBS3OMD22 (NCT07877714)
- Where
- 10 sites in Italy
- Volunteers
- 16,000 people
- Sponsor
- Istituto Giannina Gaslini
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
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
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