Clinical trial NCT02921945: Efficacy and Safety Study of SCT800 for On-demand Treatment With in Patients With Hemophilia A
Official title: A Multi-center, Phase III, Non-controlled, Open-label Trial to Evaluate the Safety and Efficacy of SCT800 for On-demand Treatment in Previously Treated Patients With Hemophilia A
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Hemophilia A
Lead sponsor: Sinocelltech Ltd.
Sponsor class: INDUSTRY
First posted: 2016-10-03
Enrollment: 50
Sex: ALL
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: In this trial safety and efficacy of SCT800 (B-domain deleted recombinant factor VIII) is being evaluated in 50 subjects, 12 to 65 years of age, with moderate to severe Hemophilia A. These subjects will receive open label treatment with SCT800 for approximately 6 months for on-demand treatment.


Eligibility criteria: Inclusion Criteria:

* 12 to 65 years old;
* The activity of the coagulation factor VIII (FVIII:C) ≤5%, and was previously treated with FVIII concentrate(s) for a minimum of 50 exposure days (EDs) prior to study entry
* Bleeding state need to be treated with FVIII;
* 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
Secondary outcomes:
measure: Haemostatic effect of SCT800 evaluated according to a predefined four grade scale: excellent, good, moderate, or no response
timeFrame: Up to 6 months
measure: The incidence rate of FVIII inhibitors (≥0.6 BU)
timeFrame: Up to 6 months
measure: The number of infusions of SCT800 required per bleeding episode
timeFrame: Up to 6 months
measure: Actual consumption of SCT800 (IU/Kg/bleed)
timeFrame: Up to 6 months
measure: AEs related to SCT800 during the clinical study
timeFrame: Up to 6 months
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: Xielan Zhao, PhD
role: PRINCIPAL_INVESTIGATOR
References: