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

• ClinicalTrials.gov Identifier: NCT03947567
• Recruitment Status: Recruiting
• First Posted: May 13, 2019
• Last Update Posted: April 28, 2020
• Sponsor: Sinocelltech Ltd.
• Information provided by (Responsible Party): Sinocelltech Ltd.

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

• 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

• 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

Contact: Renchi Yang; 13512078851; rcyang65@163.com

Contact: Feng Xu

Locations

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

• Principal Investigator: Renchi Yang; Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College

More Information

• Responsible Party: Sinocelltech Ltd.
• ClinicalTrials.gov Identifier: NCT03947567
• Other Study ID Numbers: SCT800-A401
• First Posted: May 13, 2019
• Last Update Posted: April 28, 2020
• Last Verified: May 2019

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Hemophilia A; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn