Clinical Laboratory Tests. The Company’s clinical laboratory tests are conducted in compliance in all material respects with all applicable Health Care Laws, and, to the extent applicable, the respective counterparts thereof promulgated by governmental authorities in countries outside the United States. The Company has not received notice from the FDA, HHS or other governmental authority alleging or asserting material noncompliance with any applicable Health Care Law. To the Company’s knowledge, neither the FDA nor any other governmental authority is considering such action.
Clinical Laboratory Tests. The manufacture of the Company’s clinical laboratory tests and equipment by or on behalf of the Company or its subsidiaries is being conducted in compliance in all material respects with all applicable Health Care Laws, and, to the extent applicable, the respective counterparts thereof promulgated by governmental authorities in countries outside the United States. The Company has not had any laboratory test, clinical laboratory or manufacturing site and, to the Company’s knowledge, that any of its third-party manufacturers, subject to a governmental authority (including United States Food and Drug Administration (the “FDA”)) shutdown or import or export prohibition, nor received any FDA Form 483 or other governmental authority notice of inspectional observations or deficiencies, “warning letters,” “untitled letters,” requests to make changes to the Company’s clinical laboratory tests, processes or operations, or similar correspondence or notice from the FDA or other governmental authority alleging or asserting material noncompliance with any applicable Health Care Laws. To the Company’s knowledge, neither the FDA nor any other governmental authority is considering such action.
Clinical Laboratory Tests. A summary of observed values and change from baseline will be presented for all laboratory parameters with numerical measures using descriptive statistics. Shift tables displaying low- normal-high at baseline versus low-normal-high at end of study in a 3-by-3 contingency table will be provided. For selected laboratory parameters, scatter plots of baseline versus Week 56 results, will be produced by treatment group. A laboratory value that is above or below normal range will be considered an abnormal value. For selected laboratory parameters, threshold limits of clinical concern will be defined as multiplicative factors of the normal ranges. The list of multiplicative factors for each laboratory parameter will be included in the Statistical Analysis Plan. The frequency and percentage of subjects with laboratory results above or below the normal range and threshold limits at each scheduled assessment or any time during the treatment will be summarized by treatment group.
Clinical Laboratory Tests. Blood and urine samples will be collected as indicated in the schedule of assessments in Table 4 and Table 5, for the evaluation of haematology, serum chemistry, laboratory urinalysis/microscopy, urine culture and sensitivity, and anti BTX-A antibodies. Analysis will be performed by central laboratories. Full details regarding clinical laboratory tests are contained in the study central laboratory manual and trial master file. The investigator will review the laboratory urinalysis/microscopy, urine culture and sensitivity, haematology, and serum chemistry laboratory results; document the review, and record any clinically relevant changes occurring or observed during the study in the AE section of the eCRF (see Section 8.1.2.4 for abnormal laboratory tests that should be recorded as AEs, and Section 8.2.3 regarding specific information related to laboratory urinalysis/microscopy). All clinically relevant abnormal laboratory tests occurring during the study will be repeated at appropriate intervals until they return to Baseline or to a level deemed acceptable by the investigator and the sponsor’s clinical monitor (or his/her designated representative) or until the abnormality is explained by an appropriate diagnosis (see Section 8.2.3 for specific details regarding laboratory urinalysis/microscopy abnormalities).
Clinical Laboratory Tests. A summary of observed values and change from baseline will be presented for all laboratory parameters with numerical measures. For selected laboratory parameters, scatter plots of baseline versus week 56 results, will be produced by treatment group. A laboratory value that is above or below normal range will be considered an abnormal value. For selected laboratory parameters, threshold limits of clinical concern will be defined as multiplicative factors of the normal ranges. The list of multiplicative factors for each laboratory parameter will be included in the Statistical Analysis Plan. The frequency and percentage of subjects with laboratory results above or below the normal range and threshold limits at each scheduled assessment or any time during the treatment will be summarized by treatment group.
Clinical Laboratory Tests. All clinical laboratory test results outside of the reference range will be interpreted by the investigator using the following categories: • abnormal but not a clinically significant worsening • abnormal and a clinically significant worsening
Clinical Laboratory Tests. Clinical chemistry, hematology, coagulation, and urinalysis will be presented by treatment condition and visit, summarizing the actual values and change from Baseline to each visit for each parameter using descriptive statistics for each continuous, and frequency tables for each discrete parameter. Frequency tables of predefined change abnormal of select laboratory parameter values will be generated.
Clinical Laboratory Tests. Clinical laboratory data will be listed for each subject. Values outside the normal laboratory reference ranges will be flagged as high or low on the listings.
Clinical Laboratory Tests. The tests detailed in Table 7 will be performed by the local laboratory or central laboratories as appropriate. • Protocol-specific requirements for inclusion or exclusion of participants are detailed in Section 5 of the protocol. • Additional tests may be performed at any time during the study as determined necessary by the Investigator or required by local regulations. Investigators must document their review of each laboratory safety report and indicate whether out of range results are clinically significant or not clinically significant. • Women of childbearing potential should only be enrolled after a negative serum pregnancy test result at screening. Additional urine pregnancy testing will be standard for the protocol unless serum testing is required by site policies, local regulation, or IRBs/IECs and should be performed per the time points specified in the SoA (Section 1.3). Screening pregnancy criteria are detailed in Section 5.1.
Clinical Laboratory Tests. The tests detailed in Table 5 will be performed by the central and/or local laboratory. Protocol- specific requirements for inclusion or exclusion of patients are detailed in Section 5 of the protocol. Additional tests may be performed at any time during the study as determined necessary by the Investigator or required by local regulations. Investigators must document their review of each laboratory report. Table 5: Protocol-Required Laboratory Assessments1 Complete blood count (CBC), including: - RBC count - White blood cell (WBC) count - WBC differential (absolute and percent): - neutrophils - lymphocytes - monocytes - eosinophils - basophils - Hematocrit (Hct) - Hemoglobin (Hgb) - Mean corpuscular volume (MCV) - Mean corpuscular hemoglobin (MCH) - Mean corpuscular hemoglobin concentration (MCHC) - Mean platelet volume (MPV) - Platelet count - Red cell distribution width (RDW) - Reticulocyte count (absolute and percent) Alanine aminotransferase (ALT) Albumin Alkaline phosphatase Aspartate aminotransferase (AST) Bicarbonate (HCO3) Bilirubin (fractionated)2 Blood urea nitrogen (BUN) Calcium Calculated eGFR3 Chloride C-reactive protein (CRP) Creatine kinase4 Creatinine Gamma-glutamyl transferase Glucose5 LDH Lipid profile including: - Cholesterol/HDL ratio - High-density lipoprotein cholesterol (HDL-C) - Low-density lipoprotein cholesterol (LDL-C) - Non-HDL-C - Total cholesterol - Triglycerides - Very low-density lipoprotein cholesterol (VLDL-C) Potassium Sodium Total protein Uric acid Urinalysis and microscopy: - Bilirubin - Color - Glucose - Hgb - Ketones - Leukocytes - Nitrite - Occult blood - pH - Protein - Specific gravity - Urobilinogen Microscopic examination of sediment Urine pregnancy test6 Urine drug screens: - Cocaine - Amphetamines, - Barbiturates - Benzodiazepines - Cannabinoids - Opiates - Phencyclidine - Propoxyphene - methadone Hemolytic alternative pathway activity (APH) Bb C3 C3 fragment deposition Hemolytic classical pathway activity (CH50) D-dimer Direct Xxxxxx Factor D Free hemoglobin Follicle stimulating hormone (FSH) Haptoglobin Plasma/serum samples for additional nongenetic biomarker testing PNH clone size Coagulation (PT/PTT/INR) Pregnancy (serum)6 Iron studies: - Serum ferritin - Serum iron - Serum transferrin - Transferrin saturation (TSAT) - Total iron binding capacity (TIBC) Serology: - HCV Ab - HbsAg - HbcAb - HIV Ab - UGT1A1 (Xxxxxxx’x)7 Genetic biomarkers (optional) PK
