Brief Title
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
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.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Incremental Recovery (K-value)
Secondary Outcome
Haemostatic effect of SCT800 evaluated according to a predefined four grade scale: excellent, good, moderate, or no response
Condition
Hemophilia A
Intervention
SCT800
Study Arms / Comparison Groups
SCT800
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
50
Start Date
October 2016
Completion Date
June 2018
Primary Completion Date
December 2017
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;
Gender
All
Ages
12 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Xielan Zhao, PhD, ,
Location Countries
China
Location Countries
China
Administrative Informations
NCT ID
NCT02921945
Organization ID
SCT800HA3
Responsible Party
Sponsor
Study Sponsor
Sinocelltech Ltd.
Study Sponsor
Xielan Zhao, PhD, Principal Investigator, Xiangya Hospital of Centre-South University, Changsha, China
Verification Date
September 2016