Study Endpoints. 14.2.1 Primary endpoint and analysis The primary endpoint is a demonstration that the investigational group has a higher pCR rate than the historical control group. This will be achieved using the following decision rules. In the ER-positive/HER2-negative sub-group, the trial will proceed in two stages: 16 patients will be treated in the first stage, and the treatment will be declared insufficiently active if at most 2 pCR are reported. In the second stage, a total of 26 patients will be treated, and · the treatment will be declared insufficiently active if at most 3 pCR are reported · the treatment will be declared active if at least 6 pCR are reported · the trial is inconclusive if 4 or 5 pCR are reported In the ER-negative/HER2-negative sub-group, the trial will proceed in two stages: 12 patients will be treated in the first stage, and the treatment will be declared insufficiently active if at most 4 pCR are reported. In the second stage, a total of 28 patients will be treated, and · the treatment will be declared insufficiently active if at most 11 pCR are reported · the treatment will be declared active if at least 14 pCR are reported · the trial is inconclusive if 12 or 13 pCR are reported If either of the sub-groups is inconclusive, the observed pCR rate in the other sub-group will inform the decision to further investigate the treatment in other trials. If both sub-groups are inconclusive, other considerations will inform the decision to further investigate the treatment in other trials. In each sub-group and for each analysis performed, the pCR rate of the investigational group will be calculated and reported, along with 95% Wxxxxx score confidence intervals.
14.2.2 Secondary endpoints and analyses Secondary endpoints of this study are: · Pathologic complete response (breast) · Clinical complete response (cCR) · Residual cancer burden (RCB) 0-1 index · Relationship between quantitative CELx score and pCR rate Secondary analyses will also be performed with each sub-group involving additional endpoints: pathologic complete response in the breast (ypT0/Tis), clinical complete response, residual cancer burden, CELx score (quantitative). Most of these will be performed in the same way as the primary endpoint analysis. In addition, the relationship between quantitative CELx score and pCR rate in the investigational group will be studied by logistic regression of the pCR outcomes on the CELx score. This analysis will be repeated for the categorical secon...
Study Endpoints. 16 A. Toxicity ..........................................................................................16 B. Characterization of * and *-Mobilized PBPCs in Patientswith Cirrhosis ...................................................................................17
Study Endpoints. 4.1 Primary Endpoint
Study Endpoints. 5.2.1. Safety Endpoints The safety profile of RP103 will be investigated by safety reviews conducted by the Investigator at or prior to each Monthly or Quarterly visit and through adverse event report monitoring (including attribution of treatment-emergent adverse events and serious adverse events [SAEs]), physical examination (including basic neurological and skin assessment findings), xxxxx xxxxx, ECG and clinical laboratory testing as outlined in the Schedule of Events (Appendix 14.1).
5.2.2. Pharmacokinetic Endpoints The trough plasma cysteamine concentration will be measured from samples collected 0.5 hour post dose at each study visit.
5.2.3. Pharmacodynamic Endpoints The WBC cystine content will be measured from samples collected 0.5 hour post dose at each study visit.
Study Endpoints. 7.3.1. Primary efficacy endpoint
7.3.2. Secondary efficacy endpoints
Study Endpoints. 7.3.1. Safety Endpoints
Study Endpoints. Automated methods to allow patients to understand and use their own EHR notes by simplifying, translating, and offering suggested questions.
Study Endpoints. Primary Efficacy Endpoints
Study Endpoints. Safety: • Incidence and severity of adverse events (AEs), slit-lamp exam and biomicroscopy, indirect ophthalmoscopy, BCVA, IOP, SD-OCT, fundus photography, fluorescein angiography, serum chemistry, hematology, heart rate and blood pressure, physical exam, EKG and pregnancy Bioactivity: • Change in CST and macular volume based on SD-OCT at each visit post RBM-007 treatment. • Change in CNV lesion size and activity based on fundus photography, fluorescein angiography, and OCT-angiography. • Change from baseline in BCVA
Study Endpoints. 5.1 Primary endpoints The primary endpoint of this study are: - OSDI - Number of patients with OSDI improvement > 7 points (clinically significant) For these parameters, the changes occurring in the two groups at each visit vs baseline will be evaluated.
5.2 Secondary endpoints The study's secondary endpoints are the Low- and high-tech anterior segment parameters (NIBUT, TBUT, Oxford class). For these parameters, the changes occurring in the two groups at each visit compared to baseline will also be evaluated.
5.3 Safety endpoints Safety assessments will consist of recording all adverse events, adverse device effects, and product deficiencies.
