Safety Analyses. The safety endpoints are: • AEs • Biomicroscopy findings • Device deficiencies There are no safety hypotheses planned in this study. The focus of the safety analysis will be a comprehensive descriptive assessment of occurrence of adverse events as well as the other listed parameters. All AEs occurring from the time a subject signs informed consent to study exit will be accounted for in the reporting. Safety analyses will be conducted using the safety analysis set on a treatment-emergent basis. Descriptive summaries (counts and percentages) for ocular and nonocular AEs will be presented by Medical Dictionary for Regulatory Activities Preferred Terms. AEs leading to study discontinuation, significant non-serious AEs, and SAEs will be identified. Individual subject listings will be provided, as necessary. Individual subject listings will be provided for AEs that occur after signing informed consent but prior to exposure to IP. Each biomicroscopy parameter will be tabulated by its grade. For each biomicroscopy parameter, counts and percentages of eyes that experience an increase of ≥ 2 grades from baseline (last assessment prior to study lens exposure for each period) to any subsequent visit within the same period will be presented. A supportive listing will be generated which will include all biomicroscopy data from all visits within the same period for those eyes experiencing the increase. Two listings for device deficiencies, prior to exposure of study contact lenses and treatment- emergent, will be provided. Additionally, each device deficiency category will be tabulated. No inferential testing will be done for safety analysis.
Safety Analyses. Safety analyses will be performed using the Safety Analysis Set.
Safety Analyses. The safety endpoints are: • AEs • Biomicroscopy Findings • Device Deficiencies There are no safety hypotheses planned in this study. The focus of the safety analysis will be a comprehensive descriptive assessment of occurrence of AE as well as the other listed parameters. All AEs occurring from the time a subject signs informed consent to study exit will be accounted for in the reporting. Safety analyses will be conducted using the safety analysis set on a treatment-emergent basis. Descriptive summaries (counts and percentages) for ocular and nonocular AEs will be presented by Medical Dictionary for Regulatory Activities Preferred Terms. AEs leading to study discontinuation, significant non-serious AEs, and SAEs will be identified. Individual subject listings will be provided, as necessary. Individual subject listings will be provided for AEs that occur after signing informed consent but prior to exposure to IP. Two listings for device deficiencies, prior to exposure of study contact lenses and treatment- emergent, will be provided. Additionally, each device deficiency category will be tabulated. No inferential testing will be done for safety analysis.
Safety Analyses. The safety endpoints are: AEs Biomicroscopy findings Device deficiencies There are no safety hypotheses planned in this study. The focus of the safety analysis will be a comprehensive descriptive assessment of occurrence of AE as well as the other listed parameters. Descriptive summaries (counts and percentages) for ocular and nonocular AEs will be presented by Medical Dictionary for Regulatory Activities Preferred Terms, for Completed and Discontinued sets. A listing containing details of the AEs will also be provided. Each biomicroscopy parameter will be tabulated by its grade, on Completed and Discontinued analysis sets. Frequency for each device deficiency category will be presented and a supporting listing will be provided.
Safety Analyses. All adverse events and device related adverse events will be presented as the number of subjects reporting each event.
Safety Analyses. The safety population will be based on the treated population, and will be analyzed according to the actual treatment received. Data collected on all subjects who were randomized but did not receive study drug will be listed and summarized, if appropriate.
Safety Analyses. The safety endpoints are: • AEs • Biomicroscopy Findings • Device Deficiencies There are no safety hypotheses planned in this study. The focus of the safety analysis will be a comprehensive descriptive assessment of occurrence of AE as well as the other listed parameters. Descriptive summaries (counts and percentages) for ocular and nonocular AEs will be presented by Medical Dictionary for Regulatory Activities Preferred Terms, for Completed and Discontinued sets. A listing containing details of the AEs will also be provided.
Safety Analyses. Treatment emergent adverse events (TEAEs) will be summarized by system organ class (SOC) and preferred term (PT). Non-emergent events will be recorded in the data listings. For all AE tables, the number and percentage of patients reporting AEs will be grouped by the Medical Dictionary for Regulatory Activities (MedDRA) SOC and PT. Descriptive statistics for ECG, ECHO, xxxxx xxxxx, and clinical laboratory parameters will be generated. Summary statistics for each parameter at the specific visits, as well as the change from Baseline to that visit, will also be displayed. All safety data will be presented in data listings. CCI Sarepta Therapeutics, Inc. Study 4658-204 Page 10 of 55 Footnotes for Table 2 Schedule of Events: Screening through Week 48 Functional assessments, CCI , ECG, Xxxxxx ECG, and ECHO have a ±2 weeks window a For infusion visits, xxxxx xxxxx are to be collected within approximately 30 minutes prior to infusion and approximately 5, 30, and 60 minutes after the end of the infusion. If the patient has not experienced an infusion reaction after the first year of treatment, xxxxx xxxxx may be collected only 30 minutes after the end of the infusion. b Blood samples for the safety laboratory assessments must be obtained within 2 weeks prior to the Baseline/Week 1 visit, and results must be available prior to dosing at Baseline/Week 1. If more than 2 weeks have elapsed since the collection of blood samples for the Screening safety laboratory assessments, it must be repeated. If less than 2 weeks have elapsed since the collection of blood samples for the Screening safety laboratory assessments, additional safety laboratory assessments do not need to be performed. c Patients may start dosing based on local genotyping results provided that these results fulfill the required inclusion criteria; however, all patients must undergo genetic testing to confirm the exon 51 skippable mutation and CCI . C
Safety Analyses. Safety analyses will be descriptive in nature. Summary statistics for each parameter at a specific visit, as well as the change from Baseline to that visit, will also be displayed. All safety data will be presented in the data listings.
Safety Analyses. Safety analyses will be based on the reported AEs and other clinical information. Safety analyses will be performed in AT population. The safety and tolerability of selinexor will be evaluated by means of drug-related AE reports, physical examinations, and laboratory safety evaluations. The grading of the severity of the AEs will be done according to CTCAE, v.5.0. Investigators will provide their assessment as either the AE is related or not related to study drug. Treatment-emergent AEs, SAEs, AEs of at least Grade 3 in severity, related AEs, and AEs leading to withdrawal of treatment will be summarized by cohort and in the overall safety population. Treatment-emergent AEs will be those that start or worsen on or after the first day of study treatment, through 30 days after last dose. Related AEs will be those with an Investigator determination of related to study drug.
