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. Author manuscript; available in PMC: 2017 Oct 1.
Published in final edited form as: J Neurochem. 2016 Feb 10;139(Suppl 1):346–352. doi: 10.1111/jnc.13529

Table 1. Selection of relevant ongoing clinical trials for synucleinopathies.

Information regarding clinical trials was found at clinicaltrials.gov as of January 2016.

Drug name Phase Identifier(s) Target/mechanism Condition Sponsor References
TARGETING ALPHA-SYNUCLEIN
  Active immunotherapy: vaccines
  AFFITOPE PD01A I NCT02270489 NP Early MSA Affiris Schneeberger et al. 2012, Mandler et al. 2014
  AFFITOPE PD03A I NCT02270489, NCT02267434 NP Early MSA; Early PD Affiris Schneeberger et al. 2012, Mandler et al. 2014
  Passive immunotherapy: antibodies
  BIIB054 I NCT02459886 NP Healthy participants Biogen
  PRX002 I NCT02157714 C-terminus α-syn PD Prothena Biosciences Games et al. 2014
OTHER TARGETS
  GDNF (gene therapy) I NCT01621581 Neurotrophic factor PD National Institute of Neurological Disorders and Stroke Richardson et al. 2011
  Mesenchymal stem cells I NCT02315027 Regenerative MSA Mayo Clinic Lee et al. 2012
  Sargramostim (leukine) I NCT01882010 Cytokine PD Howard Gendelman, MD
  Adipose-derived stromal stem cells II NCT01453803 Regenerative, anti-inflammatory, neurotrophic effects PD Ageless Regenerative Institute
  AZD3241 II NCT02388295 Myeloperoxidase inhibitor, microglia modulation MSA AstraZeneca Stefanova et al. 2012, Kaindlstorfer et al. 2015
  Glutathione (intranasal) II NCT02424708 Antioxidant PD Bastyr University
  N-acetylcysteine II NCT02212678, NCT01470027 Glutathione precursor, antioxidant PD University of Minnesota; Cornell University
  Nicotine (transdermal) II NCT01560754 Acetylcholine receptor agonist, neuroprotective Early PD James Boyd, MD
  Caffeine III NCT01738178 Adenosine A2A receptor antagonist, neuroprotective PD McGill University Health Center
  Epigallocatechin gallate III NCT02008721 Polyphenol, antioxidant MSA Johannes Levin, MD
  Isradipine III NCT02168842 Calcium channel blocker, neuroprotective Early PD University of Rochester

NP, not provided; MSA, multiple system atrophy; PD, Parkinson’s disease.