Table 2.
Clinical trials of miRNAs in neurodegenerative diseases.
| Study name (study date) | Current state | Study type | Subjects (age) | Purpose | Design | Interventions | n | Outcome measures | Research results |
|---|---|---|---|---|---|---|---|---|---|
| NCT02253732 (09/2015-03/2018) | Completed | Interventional | MCI AD PD HV (60-85) | Prevention | Open label | Exercise | 30 | miRNA myokines Cognitive function Motoric function |
Exercise improved MDS-UPDRS, balance, walking speed, bradykinesia, REE, and glucose metabolism and increased the expression of AMPK and the expression of PRKAA1, MGF, Sirt1, Cox7a1, MyHC2, MyHC7, and other related genes increased [208]. |
| NCT02045056 (05/2014-06/2019) | Completed | Interventional | AD (65-90) | Prevention | Double-blind placebo controlled parallel design | Gemfibrozil placebo | 72 | A E miRNA-107 Aβ1-40/1-42 FCSRT PAL |
Not yet released |
| NCT03388242 (11/2017-03/2020) | Unknown | Observational | MCI (30-80) | Prevention | Open label | No intervention | 120 | (1) Fold changes of microRNAs in the blood of patients with MCI over control people. (2) Fold changes of proteins in the blood of patients with MCI over control people | Not yet released |
| NCT04137926 (03/2020-11/2022) | Recruiting | Interventional | AD HV (50-85) | Diagnostic | Double blind | miRNA battery | 360 | (1) miRNA battery for diagnostic of MCI due to AD. (2) miRNAs for intervene of MCI due to AD | Not yet released |
| NCT01819545 (01/2012-11/2015) | Unknown | Observational | AD (60-90) | Diagnostic | Open label | No intervention | 300 | Plasma and CSF press of miRNA 107 and BACE1 mRNA | (1) The overall classification accuracy of miRNA107 to discriminate between patients with amnestic mild cognitive impairment and healthy controls was 91.9%, with sensitivity of 98.3% and specificity of 82.7% [204]. (2) Alterations in key left brain regions associated with memory, language, and emotion in aMCI that were significantly correlated with plasma expression of miRNA107 and BACE1 mRNA [203] |
| NCT04120493 (10/2019-1/2021) | Recruiting | Interventional | HD (25-65) | Treatment | Double blind | Intrastriatal rAAV5-miHTT imitation (sham) surgery | 26 | AE AMT-130 fM UHDRS Q-Motor HD-CAB MRI HADS Neuro-QoL |
Not yet released |
| NCT01992029 (06/2014-10/2015) | Terminated | Observational | Amyotrophic lateral sclerosis ALS (45-70) | Diagnostic | Open label | Clinical evaluation Muscular biopsy Lumbar puncture Blood sampling Neurological assessments Neuromuscular biopsy and lumbar puncture Blood sample Cervical spinal cord and brain MRI |
5 | miRNA expression | Not yet released |
| NCT03466723 (02/2019-06/2022) | Completed | Observational | PD (30+) | Diagnostic | Open label | Genetic: biomarker identification in Parkinson's disease | 1000 | Identification of novel circulating miRNAs as biomarkers for PD | Not yet released |
| NCT05055310 (10/2016-11/2019) | Completed | Observational | AD (60-90) | Diagnostic | Open label | Diagnostic test: miRNA test | 1000 | miRNA diagnosis validation for aMCI | Not yet released |
| NCT03217396 (11/2017-09/2027) | Recruiting | Observational | MS PD ALS AD (18-65) | Diagnostic | Open label | Procedure: lumbar puncture | 300 | Concentrations of CSF (neurofilaments, beta-amyloid, tau protein, inflammatory cytokines, and microRNAs) | In a large sample of MS patients that let-7b-5p levels in the cerebrospinal fluid (CSF) were highly correlated with a number of microRNAs implicated in MS, as well as with a variety of inflammation-related protein factors, showing specific expression patterns coherent with let-7b-5p-mediated regulation [209] |
Aβ: amyloid-beta; AD: Alzheimer's disease; MCI: mild cognitive impairment; PD: Parkinson; HV: healthy volunteers; HD: Huntington's disease; AE: adverse events; FCSRT: free and cued selective reminding test; PAL: paired associate learning; BACE1: beta-site APP cleaving enzyme1; fM: CSF mutant protein; CSF: cerebrospinal fluid; UHDRS: Unified Huntington's Disease Rating Scale; Q-Motor: quantitative motor; HD-CAB: Huntington's Disease Cognitive Assessment Battery; MRI: magnetic resonance imaging; HADS: Hospital Anxiety and Depression Scale; MDS-UPDRS: clinical disability score; REE: resting energy expenditure; MGF: mechanogrowth factor.