Efficacy and Safety of ADVATE Standard Prophylaxis to Hemophilia A
Information source: Nanjing Medical University
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Hemophilia A
Intervention: Recombinant Human Coagulation Factor VIII for injection (Drug)
Phase: N/A
Status: Not yet recruiting
Sponsored by: Nanjing Medical University
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.
Clinical Details
Official title: Evaluate Efficacy and Safety of ADVATE in the Standard Prophylaxis Treatment of Severe or Moderately Severe Hemophilia A.
Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: ABR
Secondary outcome: incidence of new target jointsPettersson score of joint magnetic resonance imaging scoring of joint Hemophilia Joint Health Score (HJHS) of joint Number of ADAVTE units QoL of patients wiht hemophilia Inhibitor rate
Eligibility
Minimum age: 2 Years.
Maximum age: 18 Years.
Gender(s): Both.
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
Locations and Contacts
Additional Information
Related publications: Astermark J, Petrini P, Tengborn L, Schulman S, Ljung R, Berntorp E. Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized. Br J Haematol. 1999 Jun;105(4):1109-13. Berntorp E, Boulyjenkov V, Brettler D, Chandy M, Jones P, Lee C, Lusher J, Mannucci P, Peak I, Rickard K, et al. Modern treatment of haemophilia. Bull World Health Organ. 1995;73(5):691-701. Review. Manco-Johnson MJ, Abshire TC, Shapiro AD, Riske B, Hacker MR, Kilcoyne R, Ingram JD, Manco-Johnson ML, Funk S, Jacobson L, Valentino LA, Hoots WK, Buchanan GR, DiMichele D, Recht M, Brown D, Leissinger C, Bleak S, Cohen A, Mathew P, Matsunaga A, Medeiros D, Nugent D, Thomas GA, Thompson AA, McRedmond K, Soucie JM, Austin H, Evatt BL. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007 Aug 9;357(6):535-44. Gringeri A, Lundin B, von Mackensen S, Mantovani L, Mannucci PM; ESPRIT Study Group. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost. 2011 Apr;9(4):700-10. doi: 10.1111/j.1538-7836.2011.04214.x. Valentino LA, Mamonov V, Hellmann A, Quon DV, Chybicka A, Schroth P, Patrone L, Wong WY; Prophylaxis Study Group. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. J Thromb Haemost. 2012 Mar;10(3):359-67. doi: 10.1111/j.1538-7836.2011.04611.x. Srivastava A, Brewer AK, Mauser-Bunschoten EP, Key NS, Kitchen S, Llinas A, Ludlam CA, Mahlangu JN, Mulder K, Poon MC, Street A; Treatment Guidelines Working Group on Behalf of The World Federation Of Hemophilia. Guidelines for the management of hemophilia. Haemophilia. 2013 Jan;19(1):e1-47. doi: 10.1111/j.1365-2516.2012.02909.x. Epub 2012 Jul 6. Nilsson IM, Berntorp E, Löfqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992 Jul;232(1):25-32. Ljung R. Prophylactic therapy in haemophilia. Blood Rev. 2009 Nov;23(6):267-74. doi: 10.1016/j.blre.2009.08.001. Epub 2009 Sep 22. Review.
Starting date: November 2014
Last updated: October 29, 2014
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