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

Primary Purpose

Hemophilia A

Status

Unknown status

Phase

Phase 3

Locations

Study Type

Interventional

Intervention

Recombinant Human Coagulation FVIII

About this trial

This is an interventional prevention trial for Hemophilia A

Eligibility Criteria

undefined - 12 Years (Child)MaleDoes not accept healthy volunteers

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;

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Recombinant Human Coagulation FVIII

Arm Description

Participant receivedSCT800 for prophylaxis with 25 - 50 IU/kg injection once every other day or three times per week for 6 months.

Outcomes

Primary Outcome Measures

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)

Secondary Outcome Measures

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).

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]

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).

Elimination Half Life

t1/2; Chromogenic Assay

Clearance

CL; Chromogenic Assay

Full Information

NCT ID

NCT03947320

First Posted

May 9, 2019

Last Updated

May 9, 2019

Sponsor

Sinocelltech Ltd.

1. Study Identification

Unique Protocol Identification Number

NCT03947320

Brief Title

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

Official Title

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.

Study Type

Interventional

2. Study Status

Record Verification Date

May 2019

Overall Recruitment Status

Unknown status

Study Start Date

December 20, 2019 (Anticipated)

Primary Completion Date

September 20, 2020 (Anticipated)

Study Completion Date

September 20, 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Sinocelltech Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

5. Study Description

Brief Summary

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hemophilia A

7. Study Design

Primary Purpose

Prevention

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

70 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Recombinant Human Coagulation FVIII

Arm Type

Experimental

Arm Description

Participant receivedSCT800 for prophylaxis with 25 - 50 IU/kg injection once every other day or three times per week for 6 months.

Intervention Type

Drug

Intervention Name(s)

Recombinant Human Coagulation FVIII

Other Intervention Name(s)

SCT800

Intervention Description

Participant received SCT800 for prophylaxis with 25 - 50 IU/kg injection once every other day or three times per week for 6 months.

Primary Outcome Measure Information:

Title

Annualized Bleeding Rate

Description

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)

Time Frame

up to 24 weeks

Secondary Outcome Measure Information:

Title

Annualized joint bleeding rate

Description

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).

Time Frame

up to 24 weeks

Title

FVIII incremental in-vivo recovery

Description

Incremental recovery is determined as the peak factor level recorded in the first hour after infusion and is reported as [IU/ml]/[IU/kg]

Time Frame

Predose within 30 min，15 min±2 min

Title

Bleeding event treatment efficacy

Description

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).

Time Frame

up to 24 weeks

Title

Elimination Half Life

Description

t1/2; Chromogenic Assay

Time Frame

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

Title

Clearance

Description

CL; Chromogenic Assay

Time Frame

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 Pre-specified Outcome Measures:

Title

Incidence of FVIII inhibitors

Description

The Nijmegen-Bethesda assay shall be used to monitor the production of FVIII inhibitors during the trial.

Time Frame

up tp 24 weeks

10. Eligibility

Sex

Male

Gender Based

Yes

Gender Eligibility Description

Hemophilia A is a kind of sex chromosome recessive genetic disease and ofter occurs in male.

Maximum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

Eligibility Criteria

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;

12. IPD Sharing Statement

Learn more about this trial

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

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