ROC analysis for sEV effectiveness prediction

Clinical Score: 0.900 Price: $0.50 cardiovascular disease human patients Status: proposed

What This Experiment Tests

Clinical experiment designed to assess clinical efficacy targeting miR-130a in human patients. Primary outcome: predictive accuracy for sEV effectiveness

Description

This clinical validation experiment used Receiver Operating Characteristic (ROC) curve analysis to evaluate the predictive capability of miR-130a and TGF-β content for identifying ineffective sEV. The study demonstrated that combining these biomarkers 'in Series' could predict sEV ineffectiveness with a Likelihood Ratio+ of 3.3 (95% CI: 2.6-3.9). This analysis provided the clinical validation needed to support the use of these biomarkers for patient selection in sEV therapy.

TARGET GENE
miR-130a
MODEL SYSTEM
human patients
ESTIMATED COST
$0
TIMELINE
0 months
PATHWAY
TGF-β signaling, angiogenesis
SOURCE
extracted_from_pmid_31959759
PRIMARY OUTCOME
predictive accuracy for sEV effectiveness

Scoring Dimensions

Info Gain 0.00 (25%) Feasibility 0.00 (20%) Hyp Coverage 0.00 (20%) Cost Effect. 0.00 (15%) Novelty 0.00 (10%) Ethical Safety 0.00 (10%) 0.900 composite

Protocol

ROC Analysis for sEV Effectiveness Prediction Protocol

Phase 1: Sample Collection and sEV Isolation (Days 1-14)

Patient Recruitment: Enroll n=120 cardiovascular disease patients (60 with acute coronary syndrome, 60 age/sex-matched stable angina controls) undergoing elective cardiac catheterization at tertiary cardiovascular center. Obtain written informed consent for sEV collection from plasma.

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Expected Outcomes

Primary Outcomes

Diagnostic Performance: Combined miR-130a/TGF-β model achieves AUC ≥ 0.82 (95% CI: 0.74-0.89) for predicting sEV therapeutic response. Single-marker miR-130a achieves AUC ≥ 0.72; single-marker TGF-β achieves AUC ≥ 0.75.

Cutpoint Optimization: Optimal sensitivity-specificity threshold yields sensitivity ≥78% and specificity ≥70% for identifying patients likely to respond to sEV therapy.

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Success Criteria

Primary Success Criteria

AUC Threshold: Multi-marker model must achieve AUC ≥ 0.80 with lower 95% CI bound ≥ 0.70 for clinical utility. Single-marker models must exceed AUC ≥ 0.70 to be considered clinically relevant.

Sensitivity/Specificity Balance: At optimal operating point, model must achieve either (a) sensitivity ≥75% with specificity ≥65%, OR (b) specificity ≥75% with sensitivity ≥65%, reflecting clinically useful bidirectional discrimination.

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