Back to results

Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients.

Primary Purpose

Hemophilia A

Status

Recruiting

Phase

Phase 4

Locations

China

Study Type

Interventional

Intervention

Recombinant Human Coagulation FVIII

About this trial

This is an interventional prevention trial for Hemophilia A

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Male patients who are diagnosed with severe (laboratory tested FVIII:C <2%) hemophilia A;
Patients Who Completed trial of SCT800-A302 or SCT800-A 303 and New PTP(previously treated patients)(Previously received FVIII prophylactic or bleeding treatment, have the relevant records and are verified to have accumulated exposures days( EDs) ≥150 days;
Negative FVIII inhibitor assay results (laboratory tested Nijmegen-Bethesda assay result <0.6 BU(Bethesda unit)/mL);
The treatment records of at least 50EDs before screening can be obtained;
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;
Patients with other coagulation dysfunction diseases in addition to hemophilia A;
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

Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Recombinant Human Coagulation FVIII

Arm Description

Outcomes

Primary Outcome Measures

Incidence of FVIII inhibitors

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

Secondary 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)

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).

Full Information

NCT ID

NCT03947567

First Posted

May 9, 2019

Last Updated

April 26, 2020

Sponsor

Sinocelltech Ltd.

1. Study Identification

Unique Protocol Identification Number

NCT03947567

Brief Title

Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients.

Official Title

A Multicenter, Open, Extension Trial to Evaluate Safety and Efficacy of Recombinant Human Coagulation Factor VIII (SCT800) During Long Term Treatment in Previously Treated Patients With Severe Haemophilia A .

Study Type

Interventional

2. Study Status

Record Verification Date

May 2019

Overall Recruitment Status

Recruiting

Study Start Date

July 26, 2019 (Actual)

Primary Completion Date

April 1, 2025 (Anticipated)

Study Completion Date

April 1, 2025 (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

Data Monitoring Committee

5. Study Description

Brief Summary

This study is a multi-center, open-label, extension trial to evaluate the safety, efficacy of SCT800 in long term prophylaxis and on-demand treatment in patients 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 V1, on-demand treatment is given when bleeding episodes occur. The study period is 120 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 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

240 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Recombinant Human Coagulation FVIII

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

Recombinant Human Coagulation FVIII

Other Intervention Name(s)

SCT800

Intervention Description

Participants received SCT800 for prophylaxis and/or on-demand treatment for 120 weeks.

Primary Outcome Measure Information:

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 to 120 weeks

Secondary 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 120 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

up to 120 weeks

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 120 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.

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Male patients who are diagnosed with severe (laboratory tested FVIII:C <2%) hemophilia A; Patients Who Completed trial of SCT800-A302 or SCT800-A 303 and New PTP(previously treated patients)(Previously received FVIII prophylactic or bleeding treatment, have the relevant records and are verified to have accumulated exposures days( EDs) ≥150 days; Negative FVIII inhibitor assay results (laboratory tested Nijmegen-Bethesda assay result <0.6 BU(Bethesda unit)/mL); The treatment records of at least 50EDs before screening can be obtained; 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; Patients with other coagulation dysfunction diseases in addition to hemophilia A; 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;

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Renchi Yang

Phone

13512078851

rcyang65@163.com

First Name & Middle Initial & Last Name or Official Title & Degree

Feng Xu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Renchi Yang

Organizational Affiliation

Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College

Official's Role

Principal Investigator

Facility Information:

Facility Name

Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College

City

Tianjin

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Renchi Yang

12. IPD Sharing Statement

Learn more about this trial

Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients.

We'll reach out to this number within 24 hrs