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Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A

Primary Purpose

Hemophilia A

Status

Completed

Phase

Phase 3

Locations

China

Study Type

Interventional

Intervention

Recombinant Human Coagulation FVIII

About this trial

This is an interventional prevention trial for Hemophilia A

Eligibility Criteria

12 Years - 65 Years (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Aged ≥12 years old and ≤65 years old;
Male patients who are clinically diagnosed with severe (laboratory tested FVIII:C <1%) hemophilia A, including historical FVIII:C <1%;
Previously received FVIII treatment (prophylactic or bleeding treatment), have the relevant records and are verified to have accumulated exposures days( EDs) ≥150 days;
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(Bethesda unit)/mL);
The prothrombin time is normal or international normalized ratio (INR) ≤1.5; 7. Platelet count ≥100 × 109/L;

Exclusion Criteria:

Known allergy to recombinant coagulation factor VIII concentrate or any excipient; known allergy to bovine, rodent or hamster bovine;
Has a history or family history of blood coagulation factor VIII inhibitor;
Clinical liver function test (glutamic-pyruvic transaminase, glutamic-pyruvic transaminase) ≥ three times the upper limit of normal (ULN) or clinical kidney function test (blood urea nitrogen, creatinine) ≥ three times the ULN;
Patients clinically diagnosed with active Hepatitis B or Hepatitis C;
Patients with other coagulation dysfunction diseases in addition to hemophilia A;
Patients with severe heart disease, including myocardial infarction and cardiac dysfunction of class III or above;
Patients who previously experienced intracranial bleeding;

Sites / Locations

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

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; One-stage aPTT Clotting Assay and Chromogenic Assay

Clearance

CL; One-stage aPTT Clotting Assay and Chromogenic Assay

Area Under the Curve to the Last Measurable Timepoint

AUClast;One-stage aPTT Clotting Assay and Chromogenic Assay

Full Information

NCT ID

NCT03815318

First Posted

January 17, 2019

Last Updated

February 4, 2020

Sponsor

Sinocelltech Ltd.

Collaborators

Chinese Academy of Medical Sciences, Parexel, Q2 Solutions

1. Study Identification

Unique Protocol Identification Number

NCT03815318

Brief Title

Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A

Official Title

A Multicenter, Open, Single Arm Evaluation of the Efficacy, Safety and Pharmacokinetics of Recombinant Human Coagulation FVIII (SCT800) in Prophylaxis Therapy on Patients With Severe Hemophilia A Who Had Previously Treated With FVIII.

Study Type

Interventional

2. Study Status

Record Verification Date

February 2020

Overall Recruitment Status

Completed

Study Start Date

January 21, 2019 (Actual)

Primary Completion Date

January 16, 2020 (Actual)

Study Completion Date

January 16, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Sinocelltech Ltd.

Collaborators

Chinese Academy of Medical Sciences, Parexel, Q2 Solutions

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, single-arm 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:C) . 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 and prophylaxis with SCT800 shall continue for 24 consecutive weeks.

Detailed Description

After subjects pass screening, prophylaxis with 25 - 50 IU/kg of SCT800 shall be administered once every other day or three times per week for 6 months. Subjects shall receive FVIII intravenous injection treatment at the study site or at home. During the study prophylaxis treatment period, subjects should return for visit every four weeks, namely for Visit (V) 2 (4 weeks ± 4 days), Visit 3 (8 weeks ± 4 days), Visit 4 (12 weeks ± 4 days), Visit 5 (16 weeks ± 7 days), Visit 6 (20 weeks ± 7 days), Visit 7 (24 weeks ± 7 days) and end-of-treatment (EOT) visit (+ 14 days). Of which, blood collection shall be carried out during the screening period and in V1, V2, V4, V7 and the EOT visit for FVIII inhibitor assay; blood collection shall be carried out before and after the completion of SCT800 infusion in V1, V4 and V7 for incremental in-vivo recovery (IVR) assay. FVIII:C activity shall be monitored before injection and 15minutes(min), 30min, 1h, 3h, 6h, 9h, 12h, 24hours(h), 28h, 32h and 48h after injection at V1 and V7. Then pharmacokinetics parameters (incremental in-vivo recovery, t1/2 etc.) of SCT800 shall be calculated and evaluated. 5 subjects may receive elective surgical treatment within the prophylaxis treatment period (after the first drug administration in day 0).

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

73 (Actual)

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、1 hour±5 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; One-stage aPTT Clotting Assay and Chromogenic Assay

Time Frame

Predose within 30 min，15 min±2 min、30 min±2 min，1 hour±5 min，3 hours±5 min，6 hours±5 min，9 hours±10 min，12hours h±10 min，24 hours±10 min，28 hours±10 min，32 hours±10 min and 48 hours±10 min post-dose.

Title

Clearance

Description

CL; One-stage aPTT Clotting Assay and Chromogenic Assay

Time Frame

Title

Area Under the Curve to the Last Measurable Timepoint

Description

AUClast;One-stage aPTT Clotting Assay and Chromogenic Assay

Time Frame

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.

Minimum Age & Unit of Time

12 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Aged ≥12 years old and ≤65 years old; Male patients who are clinically diagnosed with severe (laboratory tested FVIII:C <1%) hemophilia A, including historical FVIII:C <1%; Previously received FVIII treatment (prophylactic or bleeding treatment), have the relevant records and are verified to have accumulated exposures days( EDs) ≥150 days; 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(Bethesda unit)/mL); The prothrombin time is normal or international normalized ratio (INR) ≤1.5; 7. Platelet count ≥100 × 109/L; Exclusion Criteria: Known allergy to recombinant coagulation factor VIII concentrate or any excipient; known allergy to bovine, rodent or hamster bovine; Has a history or family history of blood coagulation factor VIII inhibitor; Clinical liver function test (glutamic-pyruvic transaminase, glutamic-pyruvic transaminase) ≥ three times the upper limit of normal (ULN) or clinical kidney function test (blood urea nitrogen, creatinine) ≥ three times the ULN; Patients clinically diagnosed with active Hepatitis B or Hepatitis C; Patients with other coagulation dysfunction diseases in addition to hemophilia A; Patients with severe heart disease, including myocardial infarction and cardiac dysfunction of class III or above; Patients who previously experienced intracranial bleeding;

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

12. IPD Sharing Statement

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Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A

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