Clinical trials: Aromatic Amino Acid Decarboxylase Deficiency
Cerebral Palsy, Genetic Disease
Genetic Etiology in Patients With Cerebral Palsy
Cerebral palsy (CP) is a permanent disorder of movement or posture due to non-progressive impairments of the developing brain. Current estimates indicate that as many as 30% of CP cases may be genetic in nature.
- Conditions
- Cerebral Palsy, Genetic Disease, Developmental Delay, Metabolism, Inborn Errors
- Trial name
- GenCP (NCT05123768)
- Testing
- NGS
- Where
- Slovenia
- Volunteers
- 150 people
- Sponsor
- University Medical Centre Ljubljana
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
Aromatic L-amino Acid Decarboxylase Deficiency
Newborn Screening for Aromatic L-amino Acid Decarboxylase Deficiency
Parents of newborns will be invited to test if their newborns are affected with Aromatic L-amino Acid Decarboxylase (AADC) deficiency.
- Trial name
- NCT02399761
- Where
- Taiwan
- Volunteers
- 154,795 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
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.
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
Aromatic Amino Acid Decarboxylase Deficiency
A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
This clinical trial expansion is to offer patients, who are not enrolled into the Phase I/II trial, a chance of treatment, to provide the experience in this gene therapy, and to increase the dose slightly.
- Trial name
- NCT02926066
- Testing
- AAV2-hAADC
- Where
- Taiwan
- Volunteers
- 12 people
- Sponsor
- National Taiwan University Hospital
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
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 Deficiency
Prevalence of High Plasmatic 3OMethyldopa Level in a Specific Population of Patients With a Symptomatology Compatible With AADC Deficiency
O-MethyDopa (3-OMD) is a metabolite of the Dopaminergic pathway that accumulates in case of a default in the neurotransmitter biosynthesis due to a key enzyme deficiency: Aromatic L-Amino Acid Decarboxylase (AADC) deficiency.
- Trial name
- DOPADEF (NCT05211609)
- Testing
- Plasmatic 3-O-Methyldopa dosage
- Where
- 2 sites in France
- Volunteers
- 388 people
- Sponsor
- University Hospital, Montpellier