Table 1.
Drug-based clinical trials targeting α-synuclein accumulation directly or indirectly.
| Molecule | Mechanism | Clinical Trial Phase | Year | Location of Trial | Reference | |
|---|---|---|---|---|---|---|
| Immunotherapy | PRX002 | Monoclonal antibody targeting C-terminal sequence of α-syn (amino acids 118–126) | 1 (healthy volunteers) | 2016 | United States | NCT02095171 [190] |
| 1 (healthy volunteers and PD patients) | 2017 | United States | NCT02157714 [191] | |||
| 2 (PD patients) | Active | United States | NCT03100149 | |||
| MEDI1341 | Monoclonal antibody targeting C-terminal sequence of α-syn | 1 (healthy volunteers) | Recruiting | United States, United Kingdom | NCT03272165 | |
| BIIB054 | Monoclonal antibody targeting N-terminal aggregated forms of α-syn | 1 (healthy volunteers and PD patients) | 2018 | United States | NCT02459886 [198] | |
| 2 (PD patients) | Recruiting | United States, Japan |
NCT03318523 NCT03716570 |
|||
| BAN0805 | Antibody targeting protofibrils of α-syn | 1 (healthy volunteers) | Recruiting | United States | NCT04127695 | |
| PD01/PD03 Affitopes | Vaccines targeting the C-terminal sequence of α-syn via small peptides | 1 and 2 (healthy volunteers and PD patients) | 2018 | Austria |
NCT01568099 NCT02216188 |
|
| Clearance | Nilotinib | Tyrosine kinase Abelson (cAbl) inhibitor | 1 (PD, PDD and DLB patients) | 2016 | United States | NCT02281474 [329] |
| 2 (PD patients) | 2019 | United States | NCT02954978 [330] | |||
| Ambroxol | Pharmacological chaperone of β-glucocerebrosidase | 2 (PDD patients) | Recruiting | Canada | NCT02914366 [348] | |
| 2 (PD patients) | 2020 | United Kingdom | NCT02941822 [349] | |||
| Small molecules | ANLE138B | Small molecule targeting oligomeric forms of α-syn | 1 (healthy volunteers) | Recruiting | United Kingdom | NCT04208152 |
| NPT200-11 | Small molecule targeting the C-terminal region of α-syn | 1 (healthy volunteers) | 2016 | United States | NCT02606682 | |
| Anti-oxidants | CoQ10 + Vitamin E | Antioxidant activity | 3 (early PD patients) | 2013 | United States | NCT00740714 |
| Metals | Deferiprone | Chelation of iron | 1 (PD patients) | 2012 | France | NCT00943748 [434] |
| 2 (PD patients) | 2019, Recruiting | France, Canada, Austria |
NCT02728843 NCT02655315 |
Abbreviations: α-syn, α-synuclein; PD, Parkinson’s Disease; DLB, Dementia with Lewy Bodies; PDD, Parkinson’s Disease Dementia.