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出境医 / 临床实验 / Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients.

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

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.

Condition or disease Intervention/treatment Phase
Hemophilia A Drug: Recombinant Human Coagulation FVIII Phase 4

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 240 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
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 .
Actual Study Start Date : July 26, 2019
Estimated Primary Completion Date : April 1, 2025
Estimated Study Completion Date : April 1, 2025
Arms and Interventions
Arm Intervention/treatment
Experimental: Recombinant Human Coagulation FVIII Drug: Recombinant Human Coagulation FVIII
Participants received SCT800 for prophylaxis and/or on-demand treatment for 120 weeks.
Other Name: SCT800
Outcome Measures
Primary Outcome Measures :
  1. Incidence of FVIII inhibitors [ Time Frame: up to 120 weeks ]
    The Nijmegen-Bethesda assay shall be used to monitor the production of FVIII inhibitors during the trial.


Secondary Outcome Measures :
  1. Annualized Bleeding Rate [ Time Frame: up to 120 weeks ]
    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)

  2. FVIII incremental in-vivo recovery [ Time Frame: up to 120 weeks ]
    Incremental recovery is determined as the peak factor level recorded in the first hour after infusion and is reported as [IU/ml]/[IU/kg]

  3. Bleeding event treatment efficacy [ Time Frame: up to 120 weeks ]
    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).


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Hemophilia A is a kind of sex chromosome recessive genetic disease and ofter occurs in male.
Accepts Healthy Volunteers:   No
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;
Contacts and Locations

Contacts
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Contact: Renchi Yang 13512078851 rcyang65@163.com
Contact: Feng Xu

Locations
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China
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College Recruiting
Tianjin, China
Contact: Renchi Yang         
Sponsors and Collaborators
Sinocelltech Ltd.
Investigators
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Principal Investigator: Renchi Yang Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Tracking Information
First Submitted Date  ICMJE May 9, 2019
First Posted Date  ICMJE May 13, 2019
Last Update Posted Date April 28, 2020
Actual Study Start Date  ICMJE July 26, 2019
Estimated Primary Completion Date April 1, 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 9, 2019) 		Incidence of FVIII inhibitors [ Time Frame: up to 120 weeks ]
The Nijmegen-Bethesda assay shall be used to monitor the production of FVIII inhibitors during the trial.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 9, 2019)
  • Annualized Bleeding Rate [ Time Frame: up to 120 weeks ]
    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 [ Time Frame: up to 120 weeks ]
    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 [ Time Frame: up to 120 weeks ]
    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).
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients.
Official Title  ICMJE 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 .
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Condition  ICMJE Hemophilia A
Intervention  ICMJE Drug: Recombinant Human Coagulation FVIII
Participants received SCT800 for prophylaxis and/or on-demand treatment for 120 weeks.
Other Name: SCT800
Study Arms  ICMJE Experimental: Recombinant Human Coagulation FVIII
Intervention: Drug: Recombinant Human Coagulation FVIII
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 9, 2019) 		240
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 1, 2025
Estimated Primary Completion Date April 1, 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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;
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Gender Eligibility Description: Hemophilia A is a kind of sex chromosome recessive genetic disease and ofter occurs in male.
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Renchi Yang 13512078851 rcyang65@163.com
Contact: Feng Xu
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03947567
Other Study ID Numbers  ICMJE SCT800-A401
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Sinocelltech Ltd.
Study Sponsor  ICMJE Sinocelltech Ltd.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Renchi Yang Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
PRS Account Sinocelltech Ltd.
Verification Date May 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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