Hemophilia A
Conditions
Brief summary
Participants will be assigned to A or B groups with a scale of 1:1 based on a prospectively randomized treatment-sequence assignment, i.e. infuse SCT800 followed by Xyntha (group A), or the alternate sequence (group B). All participants who completed the SCT800HA1 study will enter the efficacy and safety study (Protocol No.: SCT800HA3).
Sponsors
Sinocelltech Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* 12 to 65 years old; * The activity of the coagulation factor VIII (FVIII:C) ≤2%, and was previously treated with FVIII concentrate(s) for a minimum of 50 exposure days (EDs) prior to study entry * Non-bleeding state (No clinical manifestations of active hemorrhage); * Negative assays for FVIII inhibitors (\<0.6 BU/mL); * The platelet count is normal； * Normal prothrombin time or INR ≤1.5； * Given informed consent
Exclusion criteria
* Hypersensitivity to recombinant coagulation factor VIII concentrate or any of the excipients ;allergic to heterologous proteins (e.g. murine, bovine or hamster origin); * Family history or history of FVIII inhibitors (≥0.6 Bethesda Units \[BU\] mL-1); * Received an infusion of any FVIII for on-demand therapy or prophylaxis within 4 days prior to study entry (including rFVIII, plasma-derived factor VIII \[pdFVIII\], cryoprecipitate and whole blood); * Significant hepatic or renal impairment (ALT and AST ≥2×ULN; BUN and Cr≥2×ULN); * HIV seropositive; * Abnormal hemostasis from causes other than hemophilia A； * Patients with severe heart disease, including myocardial infarction, heart failure (III or higher level); * Patients who received any anticoagulant or antiplatelet therapy within one week (including NSAIDs) or need to receive an anticoagulant or antiplatelet therapy during the period of clinical trials; * Alcoholism, drug abuse, mental disorders and mental retardation; * Elective surgery planned during the process of study; * Patients who previously participated in the other clinical trials prior to study entry; * The patient or parent/legal guardian is unable or unwilling to sign an informed consent form or to comply with the requirements of clinical protocol; * Other conditions confirmed by the researchers, resulting in that patients are unable to benefit from clinical observation;
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incremental Recovery (K-value) | 1hour after the end of the infusion | One-stage aPTT Assay |
| Area Under the Plasma Concentration Versus Time Curve From 0 to 48 Hours (AUClast) | 48 hours after the end of the infusion | One-stage aPTT Assay |
| Elimination Phase Half-life (t1/2) | 48 hours after the end of the infusion | One-stage aPTT Assay |
| Factor VIII (FVIII) Clearance (CL) | 48 hours after the end of the infusion | One-stage aPTT Assay |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of inhibitors | 72 hours after the end of the infusion | — |
| Area Under the Plasma Concentration Versus Time Curve From 0 to Infinity (AUC∞) | 48 hours after the end of the infusion | One-stage aPTT Assay |
| AEs related to SCT800 during treatment and observation of the clinical study | 48 hours after the end of the infusion | — |
| FVIII Maximum Plasma Concentration (Cmax) | 3 hours after the end of the infusion | One-stage aPTT Assay |
| Mean Residence Time (MRT) | 48 hours after the end of the infusion | One-stage aPTT Assay |
| Volume of Distribution at Steady State (Vss) | 48 hours after the end of the infusion | One-stage aPTT Assay |
Countries
China
Outcome results
None listed