Multicenter, Noncontrol Study of Recombinant Coagulation Factor VIII for Prophylactic Therapy in Subjects With Hemophilia A.
Trial Snapshot
- Phase
- Phase 3
- Enrollment
- 64
- Locations
- 11
- Primary Endpoint
- Annualized joint bleeding rate
Study Overview
Brief Summary
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Prevention
- Masking
- None
Eligibility Criteria
- Ages
- 12 Years to 65 Years (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Hemophilia A.
- •FVIII:C <1%. 3）12 and 65 years old.
- •4）Has received FVIII treatment and the treatment exposure days ≥
- •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
- •Has a history or family history of blood coagulation factor VIII inhibitor.
- •Has other coagulation dysfunction diseases in addition to hemophilia A.
- •HIV positive.
- •Plan to receive surgery during the trial.
- •Has used immunomodulator within one weeks before the first dose，and less than 7 half-life periods.
- •Known to be allergic to experimental drugs or any excipients.
- •Severe anemia and need blood transfusion.
- •Serious liver or kidney damage.
- •Serious heart disease.
- •Uncontrollable hypertension.
- •Has participated in other clinical studies within one month before the first dose.
- •The researchers believe that it is not suitable for participants.
Arms & Interventions
Prophylactic therapy
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.
Intervention: Recombinant Human Coagulation FVIII (Drug)
Outcomes
Primary Outcomes
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).
Incremental Recovery of the First Dose
Time Frame: After the first dose on day 1
Peak activity of FVIII (as Cmax) measured within 1 hour after the end of injection
Secondary Outcomes
- Incremental Recovery of Duplicated Dose(up to 12 weeks, 24 weeks)
- Monthly Average Number of Bleedings(up to 24 weeks)
- Bleeding Event Treatment Efficacy(up to 24 weeks)
- The number and dose of injection of Recombinant Human Coagulation FVIII required per bleeding episode(up to 24 weeks)
- Hemophilia Joint Health Score (HJHS)(Baseline, week 24)
- Assess the impact of Recombinant Human Coagulation FVIII following Patient Reported Outcome (PRO) EQ-5D(Baseline, week 24)