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Table 2 Active clinical trials targeting distinct pathways in Parkinson’s disease patients

From: Genotype-driven therapeutic developments in Parkinson’s disease

Trial name Registration number Study design Outcomes Study participants
α-Synuclein aggregation
Impact of Bosutinib on safety, tolerability, biomarkers and clinical outcomes in dementia with lewy bodies NCT03888222 MonoC, DB, R, PC 1st: Safety and tolerability DLB patients
Single ascending dose study of MEDI1341 in healthy volunteers NCT03272165 MultiC, DB, R, PC 1st: Safety and tolerability HVs
Endosomal/lysosomal dysfunction
A study to evaluate the safety, tolerability, and pharmacokinetics of BIIB094 in adults with Parkinson's disease (REASON) NCT03976349 MultiC, DB, R, PC 1st: Safety and tolerability mPD (LRRK2), IPD patients
A study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of DNL151 in healthy volunteers NCT04557800 MultiC, DB, R, PC 1st: Safety and tolerability HVs
Study to evaluate DNL151 in subjects with Parkinson's disease NCT04056689 MultiC, DB, R, PC 1st: Safety and tolerability PD patients
Study to evaluate DNL201 in subjects with Parkinson's disease NCT03710707 MultiC, DB, R, PC 1st: Safety and tolerability mPD (LRRK2), IPD patients
Phase 1/2a clinical trial of PR001A in patients with Parkinson's disease with at least one GBA1 mutation (PROPEL) NCT04127578 MultiC, DB, R, PC 1st: Safety and tolerability mPD (GBA), IPD patients
Ambroxol as a treatment for Parkinson's disease dementia NCT02914366 MonoC, DB, R, PC 1st: ADAS-cog and ADCS-CGIC PDD patients
Mitochondrial dysfunction
Ursodeoxycholic acid as a novel disease-modifying treatment for Parkinson's disease NCT03840005 MultiC, DB, R, PC 1st: Safety and tolerability
2nd: various
IPD patients
An omics-based strategy using coenzyme
Q10 in patients with Parkinson’s disease
DRKS00015880 MultiC, DB, R, PC 1st: MDS-UPDRS-III
2nd: 31P-MRSI
mPD (Parkin and PINK1) and genetically stratified IPD patients
Nicotinamide supplementation in early Parkinson's disease (NOPARK) NCT03568968 MultiC, DB, R, PC 1st: MDS-UPDRS-III Treatment naïve PD patients
Metabolic Cofactor Supplementation in Alzheimer's disease (AD) and Parkinson's disease (PD) patients NCT04044131 MultiC, DB, R, PC 1st: assessment of cognition, ADL, and MDS-UPDRS-III IPD or AD patients
The use of vitamin K2 in patients with Parkinson's disease and mitochondrial dysfunction (PD-K2) DRKS00019932 MonoC, DB, R, PC 1st: 31P-MRSI
2nd: additional neuroimaging marker to assess mitochondrial dysfunction
mPD (Parkin and PINK1), IPD patients, and HVs
  1. In this table, active clinical trials are listed that target either α-synuclein aggregation, endosomal/lysosomal dysfunction, or mitochondrial impairment. The registration numbers are derived from clinicaltrials.gov or the German Clinical Trials Register (accessed: 12th of December 2020). 1st: primary endpoint. 2nd: secondary endpoint
  2. 31P-MRSI 31Phosphorus Magnetic Resonance Spectroscopy Imaging, ADAS-cog Alzheimer’s Disease Assessment Scale-Cognitive Subscale, ADCS-CGIC Alzheimer’s Disease Cooperative Study—Clinical Global Impression of Change, ADL activites of daily living, DB double-blind, DLB Lewy Body Dementia, PDD Parkinson’s disease dementia, GBA Glucosylceramidase Beta, HV healthy volunteers, IPD idiopathic Parkinson’s disease, LRRK2 Leucine Rich Repeat Kinase 2, MDS-UPDRS-III Movement Disorders Society Unified Parkinson’s Disease Rating Scale Subscore III, MonoC monocentric, mPD monogenic Parkinson’s disease, MultiC multicentric, PC placebo-control, PD Parkinson’s disease, PDD Parkinson’s disease dementia, PINK1 PTEN-induced kinase 1, R randomized