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

NCT03947567 | Unknown Phase 4

• 1 other identifier: SCT800-A401
• Study Type: interventional
• Target: 240
• Locations: 1 country
• Sites: 1

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.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• Incidence of FVIII inhibitors

  The Nijmegen-Bethesda assay shall be used to monitor the production of FVIII inhibitors during the trial.

  up to 120 weeks

Secondary Outcomes (3)

• Annualized Bleeding Rate

  up to 120 weeks

• FVIII incremental in-vivo recovery

  up to 120 weeks

• Bleeding event treatment efficacy

  up to 120 weeks

Study Arms (1)

Recombinant Human Coagulation FVIII

EXPERIMENTAL

Drug: Recombinant Human Coagulation FVIII

Interventions

Recombinant Human Coagulation FVIII DRUG

Participants received SCT800 for prophylaxis and/or on-demand treatment for 120 weeks.

Also known as: SCT800

Recombinant Human Coagulation FVIII

Eligibility Criteria

• 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:

• 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.

You may not qualify if:

• 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;

Sponsors & Collaborators

• Sinocelltech Ltd.: lead

Study Sites (1)

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

Tianjin, China

RECRUITING

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

Central Study Contacts

Renchi Yang

CONTACT

13512078851; rcyang65@163.com

Feng Xu

CONTACT

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 9, 2019
• First Posted: May 13, 2019
• Study Start: July 26, 2019
• Primary Completion: April 1, 2025
• Study Completion: April 1, 2025
• Last Updated: April 28, 2020

Record last verified: 2019-05

Locations

CN (1)