Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Primary |
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) |
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence at any dose that resulted in death; was life threatening; required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in congenital anomaly/birth defect. AEs included both SAEs and non-serious AEs. TEAEs were AEs occurred following the start of treatment or AEs increasing in severity during treatment. Treatment-related TEAEs were determined by the investigator. Grades 3 AEs were severe and undesirable adverse events. Grades 4 AEs were life threatening or disabling adverse events. |
Day 1 up to Day 393 |
|
| Primary |
Number of Participants With Abnormal Laboratory Findings Without Regard to Baseline Abnormality (Including Hematology, Serum Chemistry, and Urinalysis) |
Following parameters were analyzed for laboratory examination: hematology, clinical chemistry, and urinalysis. The hematology parameters and pre-defined criteria included: neutrophils (10^3/millimeter[mm]^3) <0.8*lower limit of normal (LLN), and basophils (10^3/mm^3) >1.2*upper limit of normal (ULN). The clinical chemistry parameter and pre-defined criteria included: bilirubin (milligrams [mg]/decilitre [dL]) >1.5 ULN, aspartate aminotransferase (units [U]/liter [L]) >3.0 ULN, glucose (mg/dL) >1.5*ULN. The urinalysis parameter and pre-defined criteria included: urine glucose =1, ketones (scalar) =1, urine protein =1, urine hemoglobin (scalar) =1, and hyaline casts per low power field (/LPF). Participants met criteria at any time point were included. |
Hematology and serum chemistry: Baseline, Days 1, 29, 57, 85, 169, 253, and 365 visits. Urinalysis: Baseline, Days 1, 85, 169, 253, and 365 visits. |
|
| Primary |
Number of Participants With Changes From Baseline in Vital Signs Measurements Meeting the Pre-Defined Categorical Summarization Criteria |
Following parameters were analyzed for vital sign examination: blood pressure (BP), pulse rate (PR), temperature, respiration rate. Categorical vital signs: Temperature >38.5 degree(s) Celsius (?), Supine PR: <40 or >120 beats per minute (BPM), Systolic BP: <90 mm Hg, >=30 mm Hg change from baseline, Diastolic BP: <50 mm Hg, >=20 mm Hg change from baseline. |
Baseline, Days 1, 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365 and 393 visits. |
|
| Primary |
Number of Participants With Change From Baseline in Electrocardiogram (ECG) Parameters Meeting the Pre-defined Categorical Summarization Criteria |
Baseline was defined as the average of triplicate ECG measurements collected prior to dosing on Day 1 in B7841003. Criteria for potentially clinically important changes in ECG were defined as: PR interval value >=300 millisecond (msec); PR interval baseline >200 msec and change >=25%; PR interval baseline <=200 msec and change >=50%; QRS complex value >=140 msec and change >=50%; QTcF value >=450 msec and change >=30 msec. Only the number of participants meeting pre-defined criteria was reported below. |
Baseline, Days 1 and 29 visits |
|
| Primary |
Number of Participants With Abnormalities in Physical Examination Findings |
Physical examinations were conducted by a physician, trained physician's assistant, or nurse practitioner as acceptable according to local regulation. A full physical examination included head, ears, eyes, nose, mouth, skin, heart and lung examinations, lymph nodes, gastrointestinal, musculoskeletal, and neurological systems. The limited or abbreviated physical examination was focused on general appearance, the respiratory and cardiovascular systems, as well as towards participant reported symptoms. For measuring weight, a scale with appropriate range and resolution was used and must have been placed on a stable, flat surface. Participants removed shoes, bulky layers of clothing, and jackets so that only light clothing remains. They also removed the contents of their pockets and remain still during measurement of weight. |
Day 1 to Day 393 |
|
| Primary |
Number of Participants With Injection Site Reactions |
Injection site reactions included but were not limited to: erythema, induration, ecchymosis, pain and pruritus. Grade of severity was defined as follows: Mild: Transient or mild discomfort (< 48 hours); no medical intervention/therapy required. Moderate: Mild to moderate limitation in activity - some assistance may be needed; no or minimal medical intervention/therapy required. Severe: Marked limitation in activity, some assistance usually required; medical intervention/therapy required, hospitalizations possible. |
Day 1 to Day 365, and Day 393 visit. |
|
| Secondary |
Annualized Bleeding Rate (ABR) |
The ABR was calculated as ([number of bleeding events × 365.25] / observed treatment period in days) |
Day 1 to Day 365, and Day 393 visit. Pre-Treatment summarized the data up to 6 months prior to participation in B7841003 for de novo participants and up to 6 months prior to participation in B7841002 for roll over participants. |
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