TABLE 2.
On‐going clinical trials evaluating microRNAs as diagnostic, prognostic, and/or predictive indicators
| miRNA(s) | Source | Technology | Cancer site | Study design | Clinical application(s) | Trial ID |
|---|---|---|---|---|---|---|
| Unspecified | Tissue (frozen) | Microarrays, miRNAseq | Bone and soft tissue | Discovery and testing (n = 1000 estimated) | Prognostic value in pediatric sarcomas, osteosarcomas, neuroblastomas | NCT01050296 |
| Unspecified | Tissue, blood | Microarrays | Bone marrow | Discovery and testing (n = 529 actual) | Diagnostic value of miRNA signatures in acute myeloid leukemia | NCT00900224 |
| Unspecified | Tissue, blood | Microarrays | Bone marrow | Discovery and testing (n = 735 estimated) | Diagnostic value of miRNA signatures in acute myeloid leukemia | NCT00898092 |
| miR‐10b | Tissue, Blood, CSF | RT‐qPCR? | Brain | Testing (n = 200 estimated) | Prognostic and predictive value in gliomas | NCT01849952 |
| Unspecified | Tissue (Frozen) | miRNAseq | Brain | Discovery and testing (n = 640 estimated) | Prognostic value in glioblastomas | NCT03770468 |
| Unspecified | Blood | Unspecified | Breast | Discovery and testing (n = 506 estimated) | Predictive value for recurrence risk reduction by interventional diet, exercise and vitamin D regimen | NCT02786875 |
| Unspecified | Blood | Unspecified | Breast | Discovery and testing (n = 3500 estimated) | Prognostic value in metastatic disease | NCT02338167 |
| Unspecified | Blood (Extracellular vesicles) | Unspecified | Breast | Discovery and testing (n = 370 estimated) | Prognostic and prediction for treatment response to anti‐HER2 therapy | NCT02514681 |
| Unspecified | Tissue (FFPE) | RT‐qPCR | Breast | Discovery and testing (n = 1000 estimated) | Prognostic value in early stage hormone‐receptor positive breast cancer cases | NCT02918084 |
| Unspecified | Tissue, Blood | Unspecified | Breast | Discovery (n = 217 actual) | Predictive value for treatment response to anti‐HER2 therapy in combination with PET imaging (scheduled) | NCT01957332 |
| Undisclosed | Blood (Plasma) | RT‐qPCR | Breast | Validation (n = 300 estimated) | Predictive value for treatment response to anti‐HER2 therapy | NCT02656589 |
| Unspecified | Tissue | Microarrays | Breast | Discovery and testing (n = 550 estimated) | Predictive value of miRNAs for risk stratification of lobular cancer in situ | NCT00581750 |
| 5‐miRNA, 8‐miRNA signatures | Tissue | RT‐qPCR? | Colon, rectum | Validation (n = 200 estimated) | Diagnostic value of miRNA signature for detection of lymph node metastasis | NCT04150081 |
| Unspecified | Blood (Plasma) | RT‐qPCR | Colon, rectum | Discovery and testing (n = 200 estimated) | Predictive value of miRNAs in neoadjuvant treatment of rectal cancer | NCT03962088 |
| miR‐20a‐5p,‐21,‐103a‐3p,‐106b‐5p,‐143‐5p,‐215 | Tissue | RT‐qPCR | Colon, rectum | Interventional validation (n = 430 estimated) | Prognostic value of miRNAs in Stage II colorectal cancer | NCT02466113 |
| miR‐20a‐5p,‐21,‐103a‐3p,‐106b‐5p,‐143‐5p,‐215 | Tissue | RT‐qPCR | Colon, rectum | Validation (n = 630 estimated) | Prognostic value of miRNAs in Stage II colorectal cancer | NCT02635087 |
| Unspecified | Tissue? | Unspecified | Colon, rectum | Discovery and testing (n = 1000 estimated) | Diagnostic and prognostic value of miRNAs | NCT03309722 |
| miR‐34b‐5p,‐34c‐5p,‐34c‐3p,‐184,‐375, | Blood (Plasma) | RT‐qPCR? | Endometrium | Validation (n = 443 estimated) | Diagnostic value of miRNA signature for detection of lymph node metastasis | NCT03776630 |
| Unspecified | Blood (Serum, Plasma) | Unspecified | Gastrointestinal tract | Discovery and testing (n = 6300 estimated) | Diagnostic and prognostic value in colorectal, pancreatic, and gastroesophageal cancer | NCT04363983 |
| miR‐371 | Blood (Plasma) | Unspecified | Germ cell | Testing (n = 956 estimated) | Diagnostic value for early disease detection of testicular cancer | NCT04435756 |
| Unspecified | Tissue | NGS | Germ cell | Discovery and testing (n = 200 estimated) | Diagnostic and prognostic value in testicular stromal tumors | NCT01970696 |
| Unspecified | Saliva, Blood, Tissue (FNA) | RT‐qPCR | Head and Neck | Discovery and testing (n = 462 estimated) | Diagnostic or prognostic value of miRNA signature in matched bodily fluids or tissue samples (squamous cell carcinoma) | NCT04305366 |
| Unspecified | Blood (Serum) | Unspecified | Head and Neck | Discovery (n = 370 estimated) | Prognostic value of miRNA signature in oral cancer | NCT03202810 |
| Unspecified | Blood (Exosomes) | Unspecified | Lung | Discovery (n = 200 estimated) | Predictive value of miRNA signature for treatment response to immunotherapy treatment (scheduled) | NCT04427475 |
| Unspecified | Blood (Exosomes) | Unspecified | Lung | Discovery (n = 800) | First line of screening for lung cancer detection in combination with high‐resolution CT imaging (scheduled) | NCT04629079 |
| Unspecified | Blood | Unspecified | Lung | Discovery and testing (n = 286) | Prognostic and predictive value in metastatic lung cancer | NCT03721120 |
| Unspecified | Blood | Unspecified | Lung | Discovery and testing (n = 1000 estimated) | Diagnostic and prognostic value in early stage cases | NCT03397355 |
| 24‐miRNA signature | Blood (Plasma) | Microfluidic card | Lung | Interventional, active surveillance of smokers (n = 4119 actual) | First line of screening for lung cancer detection | NCT02247453 |
| Hummingbird microRNA profile | Blood | HMBDx microRNA Test | Lung | Testing (n = 479 actual) | First line of screening for lung cancer detection in combination with low‐dose CT imaging (scheduled) | NCT03452514 |
| Unspecified | Blood (Plasma) | HTG EdgeSeq miRNA Assay | Lung | Discovery and testing (n = 900 estimated) | Predictive value of treatment response to targetable molecular alteration or immunotherapy | NCT02511288 |
| 16‐miRNA and 45‐miRNA signatures | Blood (Plasma) | Unspecified | Lung | Testing (n = 2000 estimated) | First line of screening for lung cancer detection in combination with low‐dose CT imaging | NCT04323579 |
| Unspecified | Blood (Plasma) | Unspecified | Esophagus | Discovery and testing (n = 200 estimated) | Predictive value of miRNA signature in combination with number of circulating tumor cells | NCT02812680 |
| miR‐192‐5p,‐194‐5p,‐215‐5p | Exfoliated cells, Blood (Serum) | Digital RT‐qPCR | Esophagus | Testing (n = 220 estimated) | Diagnostic value of miRNA signature for Barrett's Esophagus | NCT02464930 |
| Unspecified | Bile (Exosomes) | Digital RT‐qPCR | Esophagus | Testing (n = 220 estimated) | Diagnostic value of miRNAs for early cancer detection | NCT02464930 |
| Unspecified | Blood | Unspecified | Pancreas | Discovery and testing (n = 5000 estimated) | Diagnostic, prognostic and predictive value | NCT03311776 |
| Unspecified | Blood | Unspecified | Pancreas and Gall Bladder | Discovery and testing (n = 500 estimated) | Diagnostic value of miRNA signature compared to or in combination with other biomarkers (e.g., CA 19‐9) | NCT02531607 |
| Unspecified | Unspecified | RNAseq, nanostring, RT‐qPCR | Pancreas | Discovery and testing (n = 200) | Prognostic and predictive value in pancreatic ductal adenocarcinoma and/or neuroendocrine tumors | NCT03840460 |
| Unspecified | Blood (Serum) | miRNAseq | Pancreas | Discovery and testing (n = 629 estimated) | Predictive value of miRNA signature for malignant progression from Multiple Endocrine Neoplasia to Neuroendocrine Tumor | NCT03048266 |
| Unspecified | Tissue, Blood, Urine, Saliva | miRNAseq | Prostate | Discovery (n = 3000 estimated) | Exploratory molecular profiling studies | NCT02594202 |
| Unspecified | Blood | Unspecified | Prostate | Discovery and testing (n = 330 estimated) | Prognostic value for clinical relapse‐free survival | NCT02745587 |
| Unspecified | Blood (Exosomes) | miRNAseq | Prostate | Discovery and testing (n = 600 estimated) | Diagnostic value of exosomal miRNAs for early disease detection in combination with PSA and MR imaging | NCT03694483 |
| X chromosome‐linked miRNAs | Unspecified | Unspecified | Prostate | Discovery and testing (n = 329 actual) | Prognostic and predictive value for treatment response to hypofractionated radiotherapy | NCT01444820 |
| let‐7c,miR‐16,‐25,‐141,‐151‐3p ‐187,‐188‐5p,‐196b, ‐200c,‐200b,‐375 | Tissue | Unspecified | Prostate | Interventional, scheduled MR imaging and biopsy (n = 628 estimated) | Diagnostic value for early disease detection in combination with PSA and PHI | NCT04283032 |
| Unspecified | Urine (Exosomes) | NGS | Prostate | Discovery and validation (n = 240 estimated) | Diagnostic value of exosomal miRNAs for separating pathological significant and insignificant prostate cancer | NCT03911999 |
| miR‐200b | Blood (Plasma) | RT‐qPCR? | Ovary | Validation (n = 443 estimated) | Prognostic value of miR‐200b changes in matched pre‐ and post‐treatment samples | NCT03776630 |
| Unspecified | Tissue | NGS | Ovary | Discovery and testing (n = 200 estimated) | Diagnostic and prognostic value in ovarian stromal tumors | NCT01970696 |
| Unspecified | Blood | Unspecified | Pelvis | Discovery and testing (n = 500 estimated) | Prognostic and predictive value in cervical, endometrial, and ovarian cancer | NCT03622983 |
| Unspecified | Tissue (Frozen) | NGS, RT‐qPCR | Stomach | Discovery and validation (n = 800 estimated) | Predictive value of miRNA signature to chemotherapy treatment | NCT03253107 |
| KRAS variants (DNA) | Saliva | DNA sequencing | Various | Validation (n = 15,000 estimated) | Role of KRAS‐variant and miRNA binding site mutation in cancer risk, prevention, and treatment | NCT02253251 |
| Unspecified | Blood (Exosomes) | Unspecified | Various | Discovery and testing (n = 1000 estimated) | Diagnostic, prognostic and/or predictive value of miRNAs in combination with circulating DNA | NCT04530890 |
| Unspecified | Blood (Plasma) | Unspecified | Various | Discovery and testing (n = 388 estimated) | Diagnostic value for cancer detection in patients with serious illness but who have no organ specific symptoms | NCT01709539 |
| Unspecified | Tissue | NGS | Various | Discovery and testing (n = 300 estimated) | Predictive value of treatment response in patients who are exceptional responders | NCT02243592 |
Note: This table includes only clinical studies with a status of “Active, not recruiting,” “Recruiting,” or “Enrolling by invitation,” and with at least 200 participants by study design (estimated or actual participants). Unless otherwise noted, these are all observational prospective studies. Interventional study indicates that participants receive a treatment and/or other procedure (e.g., imaging) that is related to intended clinical application of miRNA(s) test. Unless otherwise indicated cancer type arising from cancer site is as follows: breast carcinoma from breast, colorectal adenocarcinoma from colon and/or rectum, esophageal squamous cell carcinoma from esophagus, nonsmall cell lung cancer (adenocarcinoma or squamous cell carcinoma) from lung, pancreatic adenocarcinoma from pancreas, prostatic adenocarcinoma from prostate.
Abbreviations: CSF, cerebrospinal fluid; CT, computed tomography; MR, magnetic resonance; NGS, next generation sequencing; RT‐qPCR, reverse transcription quantitative‐polymerase chain reaction; miRNAseq, small RNA sequencing; PET, positron emission tomography; PHI, prostate health index; PSA, prostate‐specific antigen.