Efficacy and Safety of ADVATE Standard Prophylaxis to Hemophilia A
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|ClinicalTrials.gov Identifier: NCT02280265|
Recruitment Status : Unknown
Verified October 2014 by Yongjun Fang,MD, Nanjing Medical University.
Recruitment status was: Not yet recruiting
First Posted : October 31, 2014
Last Update Posted : October 31, 2014
Hemophilia A is an X-linked recessive, congenital bleeding disorder caused by deficient or defective coagulation factor VIII (FVIII). Prophylaxis is recommended as the standard of care for boys with severe haemophilia by WHO and World Federation Of Hemophilia (WFH). The efficacy and safety of prophylaxis in preventing bleeds and arthropathy in patients with hemophilia has been confirmed in well-designed clinical studies.To keep the factor level above 1%, the standard dosage for patients with severe hemophilia A is 20-40 Units /kg/infusion (average 30 Units /kg) every other day or three times a week. This dosage has a very high consumption of factor, up to 5000-6000 international unit (IU)/kg/year. The high consumption of factor and cost present a major barrier to use the standard prophylaxis in many countries particularly in the developing world.
In China the majority of boys with severe hemophilia A can only pay for on-demand treatment or low-dose prophylaxis. Ao after the affordability of patients was solved and many patients will get more chance to receive standard prophylaxis.
This study is designed to evaluate the Annual Bleeding rate (ABR), joint health outcomes and QoL outcomes in subjects using ADVATE(Recombinant Human Coagulation Factor VIII for injection) standard prophylaxis under the conditions of routine practice.
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia A||Drug: Recombinant Human Coagulation Factor VIII for injection||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Evaluate Efficacy and Safety of ADVATE in the Standard Prophylaxis Treatment of Severe or Moderately Severe Hemophilia A.|
|Study Start Date :||November 2014|
|Estimated Primary Completion Date :||December 2016|
|Estimated Study Completion Date :||December 2016|
The baseline ABR will be assessed from bleeding log and clinic records from preceding year. Subjects will initially be treated standard prophylaxis(20 - 40 IU/Kg body weight every 48 ± 6 hours) with Recombinant Human Coagulation Factor VIII for injection(ADVATE) for 1 year. Subjects must be prescribed ADVATE by the treating physician. Data will be collected over a period of 2 years from the time of study enrollment. Study visits are to coincide with routinely rescheduled and emergency visits. Available data from these visits shall be transcribed onto the case report forms (CRFs).
Drug: Recombinant Human Coagulation Factor VIII for injection
Subjects will initially be treated standard prophylaxis(20 - 40 IU/Kg body weight 2 times one week) with ADVATE for 1 year.
Other Name: ADVATE
- ABR [ Time Frame: 3 months ]To evaluate the Annual Bleeding rate (ABR) in subjects using standard prophylaxis under the conditions of routine practice.
- incidence of new target joints [ Time Frame: 3 months ]Incidence of new target joints.
- Pettersson score of joint [ Time Frame: 3 months ]Status of joint health by X-ray using Pettersson score
- magnetic resonance imaging scoring of joint [ Time Frame: 6 months ]Status of joint health by magnetic resonance imaging scoring system.
- Hemophilia Joint Health Score (HJHS) of joint [ Time Frame: 3 months ]Status of joint health using HJHS
- Number of ADAVTE units [ Time Frame: 3 months ]Number of Recombinant Human Coagulation Factor VIII for injection (ADAVTE) units required for bleed cessation and Number of ADVATE infusions required for bleed cessation
- QoL of patients wiht hemophilia [ Time Frame: 3 months ]QoL assessment by Canadian Haemophilia Outcomes - Kids Life Assessment Tool (CHO-KLAT)
- Inhibitor rate [ Time Frame: 3 months ]Inhibitor rate monitoring: inhibitor will be tested at entry of study, every 3 months and at end of study.