Clinical trial NCT02888223: Pharmacokinetic Study of SCT800 in Previously Treated Patients With Hemophilia A
Official title: Pharmacokinetic Comparison of SCT800 (B-domain Deleted Recombinant Factor VIII) With Xyntha in Previously Treated Patients With Hemophilia A: a Phase I, Open-label, Randomized, Crossover Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Hemophilia A
Lead sponsor: Sinocelltech Ltd.
Sponsor class: INDUSTRY
First posted: 2016-09-02
Enrollment: 16
Sex: ALL
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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).


Eligibility criteria: 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;

Primary outcomes:
measure: Incremental Recovery (K-value)
timeFrame: 1hour after the end of the infusion
measure: Area Under the Plasma Concentration Versus Time Curve From 0 to 48 Hours (AUClast)
timeFrame: 48 hours after the end of the infusion
measure: Elimination Phase Half-life (t1/2)
timeFrame: 48 hours after the end of the infusion
measure: Factor VIII (FVIII) Clearance (CL)
timeFrame: 48 hours after the end of the infusion
Secondary outcomes:
measure: Area Under the Plasma Concentration Versus Time Curve From 0 to Infinity (AUC∞)
timeFrame: 48 hours after the end of the infusion
measure: FVIII Maximum Plasma Concentration (Cmax)
timeFrame: 3 hours after the end of the infusion
measure: Mean Residence Time (MRT)
timeFrame: 48 hours after the end of the infusion
measure: Volume of Distribution at Steady State (Vss)
timeFrame: 48 hours after the end of the infusion
measure: Incidence of inhibitors
timeFrame: 72 hours after the end of the infusion
measure: AEs related to SCT800 during treatment and observation of the clinical study
timeFrame: 48 hours after the end of the infusion
Other outcomes:
Locations:
city: Changsha
country: China
facility: Xiangya Hospital of Centre-South University
state: Hunan
Study officials:
affiliation: Xiangya Hospital of Centre-South University, Changsha, China
name: Xie lan Zhao, PhD
role: PRINCIPAL_INVESTIGATOR
References: