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

NCT03815318 | Completed Phase 3

• 1 other identifier: SCT800-A 302
• Study Type: interventional
• Target: 73
• Locations: 1 country
• Sites: 1

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.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• 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)

  up to 24 weeks

Secondary Outcomes (6)

• Annualized joint bleeding rate

  up to 24 weeks

• FVIII incremental in-vivo recovery

  Predose within 30 min，15 min±2 min、1 hour±5 min.

• Bleeding event treatment efficacy

  up to 24 weeks

• Elimination Half Life

  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.

• Clearance

  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.

Other Outcomes (1)

• Incidence of FVIII inhibitors

  up tp 24 weeks

Study Arms (1)

Recombinant Human Coagulation FVIII

EXPERIMENTAL

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

Interventions

Recombinant Human Coagulation FVIII DRUG

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

Also known as: SCT800

Recombinant Human Coagulation FVIII

Eligibility Criteria

• Age: 12 Years - 65 Years
• Gender Eligibility Details: Hemophilia A is a kind of sex chromosome recessive genetic disease and ofter occurs in male.
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;

You may not qualify if:

• 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;

Sponsors & Collaborators

• Sinocelltech Ltd.: lead
• Chinese Academy of Medical Sciences: collaborator
• Parexel: collaborator
• Q2 Solutions: collaborator

Study Sites (1)

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

Tianjin, China

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Renchi Yang

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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 17, 2019
• First Posted: January 24, 2019
• Study Start: January 21, 2019
• Primary Completion: January 16, 2020
• Study Completion: January 16, 2020
• Last Updated: February 6, 2020

Record last verified: 2020-02

Locations

CN (1)