ClinicalTrials.gov
ClinicalTrials.gov Menu

Efficacy and Safety of ADVATE Standard Prophylaxis to Hemophilia A

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
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
Sponsor:
Information provided by (Responsible Party):
Yongjun Fang,MD, Nanjing Medical University

Brief Summary:

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)
Primary Purpose: Treatment
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


Arm Intervention/treatment
Experimental: ADVATE
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




Primary Outcome Measures :
  1. ABR [ Time Frame: 3 months ]
    To evaluate the Annual Bleeding rate (ABR) in subjects using standard prophylaxis under the conditions of routine practice.


Secondary Outcome Measures :
  1. incidence of new target joints [ Time Frame: 3 months ]
    Incidence of new target joints.

  2. Pettersson score of joint [ Time Frame: 3 months ]
    Status of joint health by X-ray using Pettersson score

  3. magnetic resonance imaging scoring of joint [ Time Frame: 6 months ]
    Status of joint health by magnetic resonance imaging scoring system.

  4. Hemophilia Joint Health Score (HJHS) of joint [ Time Frame: 3 months ]
    Status of joint health using HJHS

  5. 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

  6. QoL of patients wiht hemophilia [ Time Frame: 3 months ]
    QoL assessment by Canadian Haemophilia Outcomes - Kids Life Assessment Tool (CHO-KLAT)

  7. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   2 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Subject has hemophilia A with FVIII≤2 %.
  2. Previously treated patients (PTPs).
  3. Age from 2 to 18 years.

Exclusion Criteria:

  1. Subject has known hypersensitivity to the active substance or any of the excipients.
  2. Subject has known allergic reaction to mouse or hamster proteins.
  3. Subject has participated in another clinical study involving an investigational product (IP) or device within 30 days prior to study enrollment or is scheduled to participate in another clinical study involving another FVIII concentrate or device during the course of this study.
  4. Subject is planned, or likely to have surgery during the study period.
  5. Subject has end-stage renal failure or evidence of a severe or uncontrolled systemic disease as judged by the investigator.
  6. Subject has full-blown Acquired Immuno Deficiency Syndrome (AIDS),determined by Cluster Determinant 4+(CD4+) and clinical presentation.
  7. Subject has active hepatic disease (alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels > 5 times the upper limit of normal).
  8. Subject has clinical or laboratory evidence of severe liver impairment including (but not limited to) a recent and persistent international normalized ratio (INR)> 1.4, and/or the presence of splenomegaly and/or significant spider angioma on physical exam, and/or a history of esophageal hemorrhage or documented esophageal varices.
  9. The subject in the opinion of the investigator is unable or unwilling to comply with study protocol
  10. Subject is a family member of the investigator or site staff

Publications:

Responsible Party: Yongjun Fang,MD, Director of Department of Hematology/Oncology, Nanjing Medical University
ClinicalTrials.gov Identifier: NCT02280265     History of Changes
Other Study ID Numbers: Prophylaxis201407008-1
First Posted: October 31, 2014    Key Record Dates
Last Update Posted: October 31, 2014
Last Verified: October 2014

Keywords provided by Yongjun Fang,MD, Nanjing Medical University:
hemophilia A
standard prophylaxis
Recombinant Human Coagulation Factor VIII for injection
ADVATE
Pediatric

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants