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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