Safety,Efficacy and Pharmacokinetics Evaluation of SCT800 in Previously Treated Paediatric Patients With Severe Haemophilia A.

A Multicenter Phase III Uncontrolled Open-label Trial to Evaluate Safety and Efficacy and Pharmacokinetics of Recombinant Human Coagulation Factor VIII (SCT800) in Previously Treated Paediatric Patients With Severe Haemophilia A.

This study is a multicenter phase III uncontrolled open-label trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (<12 years old) with severe hemophilia A who have been previously treated with coagulation factor VIII(FVIII) . This study includes two phases: the screening period and prophylaxis period.Prophylaxis with 25 - 50 IU/kg of SCT800 shall be administered once every other day or three times per week starting from Visit 1 and prophylaxis with SCT800 shall continue for 24 consecutive weeks.

Study Overview

• Status: Unknown
• Conditions: Hemophilia A
• Intervention / Treatment: Drug: Recombinant Human Coagulation FVIII

• Study Type: Interventional
• Enrollment (Anticipated): 70
• Phase: Phase 3

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 12 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

Inclusion Criteria:

• Aged <12 years ;
• Male severe (central laboratory tested FVIII:C <1%) hemophilia A patients;
• Previously received FVIII treatment (prophylactic or bleeding treatment), have the relevant records and are verified to have accumulated EDs ≥150 days（6≤age<12years old）and EDs >50 days（age <6 years old）;
• The bleeding treatment records of at least 3 months before screening can be obtained;
• Negative FVIII inhibitor assay results (laboratory tested Nijmegen-Bethesda assay result <0.6 BU/mL);
• HIV negative; if HIV positive, the viral load <200 particles/uL or <400,000 copies/mL, and HIV patients must satisfy CD4+ count >200/μL;
• The patient or his guardian voluntarily signed the Informed Consent Form.

Exclusion Criteria:

• Known allergy to any coagulation factor VIII or any excipient; known allergy to bovine, rodent or hamster bovine;
• Has a history or family history of blood coagulation factor VIII inhibitor;
• Platelet count <100 × 109/L;
• Clinical liver function test ((glutamic-pyruvic transaminase, glutamic-pyruvic transaminase) ≥ five times the upper limit of normal (ULN) or clinical kidney function test （creatinine) ≥ two times the ULN;
• International normalized ratio (INR) >1.5;
• Patients with other coagulation dysfunction diseases in addition to hemophilia A;
• Patients who used any anticoagulant or anti-platelet treatment (including non-steroidal anti-inflammatory drugs [NSAIDs]) within 1 weeks before the first drug administration or who regularly (e.g., daily, every other day) use anticoagulant or anti-platelet treatment within the clinical trial period;
• Patients who used immunomodulator(e.g., immunoglobulin, corticosteroids,alpha-interferon, prednisone [>10 mg/day and >7 days], or comparable drugs, other than anti-retroviral chemotherapy) within two weeks before the first administration of the study drug or during the clinical trial period;
• Administration of any cryoprecipitate, whole blood or plasma within 30 days prior to administration of study drug.
• Patients with other clinically significant diseases, alcoholism, drug abuse, mental disorders or intellectual disabilities;
• Patients with other severe or clinical significant diseases verified by the investigator to be unable to benefit from the clinical study;
• Patients who participated in other clinical studies within one month before the first drug administration (except FVIII trials) and patients who participated in other FVIII clinical trials after signing the Informed Consent Form;

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• 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: Recombinant Human Coagulation FVIII; Participant receivedSCT800 for prophylaxis with 25 - 50 IU/kg injection once every other day or three times per week for 6 months.	Drug: Recombinant Human Coagulation FVIII; Participant received SCT800 for prophylaxis with 25 - 50 IU/kg injection once every other day or three times per week for 6 months.; Other Names: SCT800

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized Bleeding Rate	Annualized Bleeding Rate（ABR） can be calculated using the following formula: Number of bleeding events in efficacy evaluation period/(number of days in treatment period/365.25)	up to 24 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized joint bleeding rate	Annualized joint bleeding rate（AJBR） can be calculated using the following formula: Number of joint bleeding events during efficacy evaluation period/(number of days in treatment period/365.25).	up to 24 weeks
FVIII incremental in-vivo recovery	Incremental recovery is determined as the peak factor level recorded in the first hour after infusion and is reported as [IU/ml]/[IU/kg]	Predose within 30 min，15 min±2 min
Bleeding event treatment efficacy	The investigator shall evaluate the hemostatic effect after the treatment of every bleeding event of subjects based on a four-point scale(excellent, good, moderate, not relieved).	up to 24 weeks
Elimination Half Life	t1/2; Chromogenic Assay	Predose within 30 min，15 min±2 min、1 hour±5 min，10 hours±30 min，24 hours±1hours and 48 hours±2 hours post-dose
Clearance	CL; Chromogenic Assay	Predose within 30 min，15 min±2 min、1 hour±5 min，10 hours±30 min，24 hours±1hours and 48 hours±2 hours post-dose

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of FVIII inhibitors	The Nijmegen-Bethesda assay shall be used to monitor the production of FVIII inhibitors during the trial.	up tp 24 weeks

Collaborators and Investigators

• Sponsor: Sinocelltech Ltd.

Study record dates

Study Major Dates

• Study Start (Anticipated): December 20, 2019
• Primary Completion (Anticipated): September 20, 2020
• Study Completion (Anticipated): September 20, 2020

Study Registration Dates

• First Submitted: May 9, 2019
• First Submitted That Met QC Criteria: May 9, 2019
• First Posted (Actual): May 13, 2019

Study Record Updates

• Last Update Posted (Actual): May 13, 2019
• Last Update Submitted That Met QC Criteria: May 9, 2019
• Last Verified: May 1, 2019

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: SCT800-A303

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No