Safety, Efficacy Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A.

CTTQ

Status and phase: Unknown; Phase 3

Conditions: Hemophilia A

Treatments: Drug: Recombinant Human Coagulation FVIII

Study type: Interventional

Funder types: Industry

Identifiers

NCT04061109

CTTQ-NXBYZ-02

Details and patient eligibility

About

This is a multi-center, open-label, single-arm trial to evaluate the efficacy and safety of Recombinant Coagulation Factor VIII in patients with hemophilia A . Recombinant Coagulation Factor VIII is prophylactic administrated 25 - 35 IU/kg once every other day or three times per week which should be continuous for 24 weeks.

Enrollment: 64 estimated patients

Sex: All

Ages: 12 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Hemophilia A.
2. FVIII:C <1%. 3）12 and 65 years old.

4）Has received FVIII treatment and the treatment exposure days ≥100. 5）Has bleeding treatment records of at least 3 months before randomization. 6）FVIII inhibitor assay results is negative. 7） Subjects should agree to use an adequate method of contraception during the study.

8）Understood and Signed an informed consent form.

Exclusion criteria

1. Has a history or family history of blood coagulation factor VIII inhibitor.
2. Has other coagulation dysfunction diseases in addition to hemophilia A.
3. HIV positive.
4. Plan to receive surgery during the trial.
5. Has used immunomodulator within one weeks before the first dose，and less than 7 half-life periods.
6. Known to be allergic to experimental drugs or any excipients.
7. Severe anemia and need blood transfusion.
8. Serious liver or kidney damage.
9. Serious heart disease.
10. Uncontrollable hypertension.
11. Has participated in other clinical studies within one month before the first dose.
12. The researchers believe that it is not suitable for participants.

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

64 participants in 1 patient group

Prophylactic therapy

Experimental group

Description:

Subjects received Recombinant Human Coagulation FVIII for prophylactic therapy with 25 - 35 IU/kg injection once every other day or three times per week for 6 months.

Treatment:

Drug: Recombinant Human Coagulation FVIII