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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03815318
Recruitment Status : Completed
First Posted : January 24, 2019
Last Update Posted : February 6, 2020
Sponsor:
Collaborators:
Cancer Institute and Hospital, Chinese Academy of Medical Sciences
Parexel
Q2 Solutions
Information provided by (Responsible Party):
Sinocelltech Ltd.

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.

Condition or disease Intervention/treatment Phase
Hemophilia A Drug: Recombinant Human Coagulation FVIII Phase 3

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).

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 73 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
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.
Actual Study Start Date : January 21, 2019
Actual Primary Completion Date : January 16, 2020
Actual Study Completion Date : January 16, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

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 Name: SCT800




Primary Outcome Measures :
  1. Annualized Bleeding Rate [ Time Frame: up to 24 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)


Secondary Outcome Measures :
  1. Annualized joint bleeding rate [ Time Frame: up to 24 weeks ]
    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).

  2. FVIII incremental in-vivo recovery [ Time Frame: Predose within 30 min,15 min±2 min、1 hour±5 min. ]
    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 24 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).

  4. Elimination Half Life [ 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. ]
    t1/2; One-stage aPTT Clotting Assay and Chromogenic Assay

  5. Clearance [ 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. ]
    CL; One-stage aPTT Clotting Assay and Chromogenic Assay

  6. Area Under the Curve to the Last Measurable Timepoint [ 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. ]
    AUClast;One-stage aPTT Clotting Assay and Chromogenic Assay


Other Outcome Measures:
  1. Incidence of FVIII inhibitors [ Time Frame: up tp 24 weeks ]
    The Nijmegen-Bethesda assay shall be used to monitor the production of FVIII inhibitors during the trial.



Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years to 65 Years   (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:

  • 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;

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03815318


Locations
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China
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Tianjin, China
Sponsors and Collaborators
Sinocelltech Ltd.
Cancer Institute and Hospital, Chinese Academy of Medical Sciences
Parexel
Q2 Solutions
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
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Responsible Party: Sinocelltech Ltd.
ClinicalTrials.gov Identifier: NCT03815318    
Other Study ID Numbers: SCT800-A 302
First Posted: January 24, 2019    Key Record Dates
Last Update Posted: February 6, 2020
Last Verified: February 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn