Efficacy and Safety Study of SCT800 for On-demand Treatment With in Patients With Hemophilia A

Hemophilia A Clinical Trial

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT02921945
• Other study ID #: SCT800HA3
• Status: Not yet recruiting
• Phase: Phase 3
• First received: September 28, 2016
• Last updated: September 29, 2016
• Start date: October 2016
• Est. completion date: June 2018

Study information

• Verified date: September 2016
• Source: Sinocelltech Ltd.
• Contact: n/a
• Is FDA regulated: No
• Health authority: China: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 50
• Est. completion date: June 2018
• Est. primary completion date: December 2017
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years to 65 Years

Eligibility

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;

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Biological:
• SCT800
recombinant human coagulation factor VIII

Locations

Country	Name	City	State
China	Xiangya Hospital of Centre-South University	Changsha	Hunan

Sponsors (1)

Lead Sponsor	Collaborator
Sinocelltech Ltd.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incremental Recovery (K-value)	One-stage aPTT Assay	1hour after the end of the infusion	No
Secondary	Haemostatic effect of SCT800 evaluated according to a predefined four grade scale: excellent, good, moderate, or no response		Up to 6 months	No
Secondary	The incidence rate of FVIII inhibitors (=0.6 BU)		Up to 6 months	Yes
Secondary	The number of infusions of SCT800 required per bleeding episode		Up to 6 months	No
Secondary	Actual consumption of SCT800 (IU/Kg/bleed)		Up to 6 months	No
Secondary	AEs related to SCT800 during the clinical study		Up to 6 months	Yes