Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02716194 | BAX 826 Dose-Escalation Safety Study | PHASE1 | COMPLETED | 40 | — | — | Mar 3, 2016 | Jan 17, 2017 | May 25, 2021 | 28 | Bulgaria, Germany +7 |
Serious Adverse Events and non-serious Adverse Events the occurred after infusion with BAX 826.
Clinically significant results after treatment with investigational product that constitute an AE are counted. Vital signs include body temperature, respiratory rate, pulse rate, and blood pressure. Clinical laboratory results include: Hematology (hemoglobin, hematocrit, red blood cell count, white blood cell count with differential (i.e. basophils, eosinophils, lymphocytes, monocytes and neutrophils), international normalized ratio (INR), mean corpuscular volume (MCV), mean corpuscular hemoglobin concentration (MCHC), platelet count. Clinical Chemistry: sodium, potassium, chloride, bicarbonate, total protein, albumin, alanine aminotransferase (ALT), aspartate aminotransferase (AST), total bilirubin, alkaline phosphatase, gamma-glutamyltransferase (GGT), blood urea nitrogen (BUN), creatinine, glucose. Lipid panel: cholesterol, very-low-density lipoprotein (VLDL), low-density lipoprotein (LDL), high-density lipoprotein (HDL), triglycerides
Inhibition of FVIII activity by antibodies binding to FVIII were measured using the Nijmegen modification of the Bethesda inhibitor assay.
Binding antibodies to PSA FVIII (ie BAX 826) IgG and IgM
Binding antibodies to FVIII IgG and IgM
Binding antibodies to PSA (IgG and IgM)
Binding antibodies to CHO
Binding antibodies HAMA (IgG)
| Arm | Type | Description |
|---|---|---|
| Cohort 1 - Low dose | EXPERIMENTAL | The study is comprised of 3 dose cohorts and two dose escalation steps \[Cohort 1: 10 evaluable participants; Cohort 2: 10 evaluable participants; Cohort 3: 10 evaluable participants\]. Participants will be recruited to the next dose level only after short-term safety has been reviewed and subject to approval by a Safety Review Committee at the preceding dose level. |
| Cohort 2 - Medium dose | EXPERIMENTAL | The study is comprised of 3 dose cohorts and two dose escalation steps \[Cohort 1: 10 evaluable participants; Cohort 2: 10 evaluable participants; Cohort 3: 10 evaluable participants\]. Participants will be recruited to the next dose level only after short-term safety has been reviewed and subject to approval by a Safety Review Committee at the preceding dose level. |
| Cohort 3 - High dose | EXPERIMENTAL | The study is comprised of 3 dose cohorts and two dose escalation steps \[Cohort 1: 10 evaluable participants; Cohort 2: 10 evaluable participants; Cohort 3: 10 evaluable participants\]. Participants will be recruited to the next dose level only after short-term safety has been reviewed and subject to approval by a Safety Review Committee at the preceding dose level. |
| Name | Type | Description |
|---|---|---|
| BAX 826 | BIOLOGICAL | - |
| Octocog alfa | BIOLOGICAL | - |
Inclusion Criteria: 1. Previously treated male participants aged 18 to 65 years (inclusive) at the time of screening 2. Diagnosis of severe hemophilia A (Factor VIII level \<1%) 3. Previously treated with FVIII concentrates for ≥150 documented Exposure Days (EDs) 4. Karnofsky performance score of ≥...