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

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

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 Overview

• Status: Unknown
• Conditions: Hemophilia A
• Intervention / Treatment: Biological: SCT800

• Study Type: Interventional
• Enrollment (Anticipated): 50
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Hunan
    • Changsha, Hunan, China, 410008
      • Xiangya Hospital of Centre-South University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 65 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: SCT800	Biological: SCT800; recombinant human coagulation factor VIII

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incremental Recovery (K-value)	One-stage aPTT Assay	1hour after the end of the infusion

Secondary Outcome Measures

Outcome Measure	Time Frame
Haemostatic effect of SCT800 evaluated according to a predefined four grade scale: excellent, good, moderate, or no response	Up to 6 months
The incidence rate of FVIII inhibitors (≥0.6 BU)	Up to 6 months
The number of infusions of SCT800 required per bleeding episode	Up to 6 months
Actual consumption of SCT800 (IU/Kg/bleed)	Up to 6 months
AEs related to SCT800 during the clinical study	Up to 6 months

Collaborators and Investigators

• Sponsor: Sinocelltech Ltd.
• Investigators: Principal Investigator: Xielan Zhao, PhD, Xiangya Hospital of Centre-South University, Changsha, China

Study record dates

Study Major Dates

• Study Start: October 1, 2016
• Primary Completion (Anticipated): December 1, 2017
• Study Completion (Anticipated): June 1, 2018

Study Registration Dates

• First Submitted: September 28, 2016
• First Submitted That Met QC Criteria: September 29, 2016
• First Posted (Estimate): October 3, 2016

Study Record Updates

• Last Update Posted (Estimate): October 3, 2016
• Last Update Submitted That Met QC Criteria: September 29, 2016
• Last Verified: September 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: SCT800HA3

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO