5 completed, 3 recruiting, 2 active not recruiting, 1 unknown. 4 Phase 2, 2 Phase 1, 1 Early Phase 1, 1 Not applicable, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Active not recruiting 2, Completed 2 |
| Phase 1 | 2 | Completed 1, Recruiting 1 |
| Early Phase 1 | 1 | Recruiting 1 |
| Not applicable | 1 | Unknown 1 |
| Phase 3 | 1 | Active not recruiting 1 |
Follow Aromatic amino acid decarboxylase deficiency: new trials by email Literature Review of Aromatic amino acid decarboxylase deficiency trials Research Report on Aromatic amino acid decarboxylase deficiency
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency VGN-R09b injection - Aromatic L-amino Acid Decarboxylase Deficiency | Phase 2 / Phase 3 | Active not recruiting | Shanghai Vitalgen BioPharma Co., Ltd. | 13 | 2024-07-02 | 2026-04-21 |
| Newborn Screening and Early Diagnosis of AADC Deficiency in the Ligurian Population Neurology, Aromatic Amino Acid Decarboxylase Deficiency | Recruiting | Istituto Giannina Gaslini | 16000 | 2023-08-17 | 2026-10-16 | |
| An Early Clinical Trial to Evaluate VGN-R09b for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency. VGN-R09b - Aromatic L-amino Acid Decarboxylase (AADC) Deficiency | Early Phase 1 | Recruiting | Shanghai Jiao Tong University School of Medicine | 6 | 2023-01-30 | 2023-03-13 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Prevalence of High Plasmatic 3OMethyldopa Level in a Specific Population of Patients With a Symptomatology Compatible With AADC Deficiency Plasmatic 3-O-Methyldopa dosage - Aromatic L-Amino Acid Decarboxylase Deficiency | Not applicable | Unknown | University Hospital, Montpellier | 388 | 2022-05-20 | 2022-08-11 |
| A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants Eladocagene Exuparvovec - AADC Deficiency | Phase 2 | Active not recruiting | PTC Therapeutics | 13 | 2021-05-12 | 2026-04-24 |
| Genetic Etiology in Patients With Cerebral Palsy NGS - Cerebral Palsy, Genetic Disease | Completed | University Medical Centre Ljubljana | 150 | 2021-04-01 | 2022-11-02 | |
| A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion AAV2-hAADC - Aromatic Amino Acid Decarboxylase Deficiency | Phase 2 | Completed | National Taiwan University Hospital | 12 | 2016-11-09 | 2023-02-10 |
| A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients AAV2-hAADC - AADC Deficiency | Phase 1 | Recruiting | Krzysztof Bankiewicz | 42 | 2016-07-01 | 2026-08-27 |
| A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC gene therapy - Aromatic L-amino Acid Decarboxylase (AADC) Deficiency | Phase 1 / Phase 2 | Completed | National Taiwan University Hospital | 10 | 2014-10-22 | 2023-02-10 |
| Newborn Screening for Aromatic L-amino Acid Decarboxylase Deficiency Aromatic L-amino Acid Decarboxylase Deficiency | Completed | National Taiwan University Hospital | 154795 | 2012-08-01 | 2017-04-28 |
3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency (1) · Acidemia, isovaleric (1) · Acyl-CoA Dehydrogenase Family, Member 9, Deficiency of (1) · Adrenal Hyperplasia, Congenital (1) · Adrenoleukodystrophy (1) · Andersen Syndrome (1) · Anemia, Diamond-Blackfan (1) · Anemia, Sickle Cell (1) · Argininosuccinic Aciduria (1) · Aspartylglucosaminuria (1) · Ataxia (1) · Ataxia with vitamin E deficiency (1) · Basal ganglia disease, biotin-responsive (1)
Brain Diseases (2) · Central Nervous System Diseases (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Metabolic Diseases (2) · Musculoskeletal Diseases (2) · Nervous System Diseases (2) · Neurobehavioral Manifestations (2) · Neurologic Manifestations (2) · Nutritional and Metabolic Diseases (2) · Pathological Conditions, Signs and Symptoms (2) · Abnormalities, Multiple (1) · Adrenal Gland Diseases (1) · Adrenal Insufficiency (1) · Adrenogenital Syndrome (1) · Albinism (1)
Genetic Therapy (1)
National Taiwan University Hospital (3) · Centre Hospitalier Universitaire de Liege (1) · Istituto Giannina Gaslini (1) · Krzysztof Bankiewicz (1) · PTC Therapeutics (1) · Shanghai Jiao Tong University School of Medicine (1) · Shanghai Vitalgen BioPharma Co., Ltd. (1) · University Hospital, Montpellier (1) · University Medical Centre Ljubljana (1)
Taiwan (4) · China (2) · United States (2) · Belgium (1) · France (1) · Israel (1) · Italy (1) · Slovenia (1)
Jiwen Wang, Doctor (1) · Krystof Bankiewicz, MD, PhD (1) · Laurent Servais (1) · Wuh-Liang Hwu, M.D., Ph.D. (1) · Yin-Hsiu Chien, MD.,PhD (1)