Ignite Creation Date:
2024-05-06 @ 3:24 AM
Last Modification Date:
2024-10-26 @ 11:33 AM
Study NCT ID:
NCT02282410
Status:
UNKNOWN
Last Update Posted:
2014-11-04
First Post:
2014-10-29
Brief Title:
Evaluate Efficacy and Safety of ADVATE in the Standard Prophylaxis Treatment of Severe or Moderately Severe Hemophilia A
Sponsor:
The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
Organization:
The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
Study Overview
Official Title:
Evaluate Efficacy and Safety of ADVATE in the Standard Prophylaxis Treatment of Severe or Moderately Severe Hemophilia A
Status:
UNKNOWN
Status Verified Date:
2014-10
Last Known Status:
NOT_YET_RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Based on the successful pioneer work of Dr Nilsson and her colleagues in Sweden that started in the late 1950s prophylaxis is recommend 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 studiesTo keep the factor level above 1 the standard dosage for patients with severe hemophilia A is 20-40 Units kginfusion 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 unitIUkgyear The high consumption of factor and cost present a major barrier to use the standard prophylaxis in many countries particularly in the developing world
But after the Recombinant Human Coagulation Factor VIII for injection ADVATE Patient Assistance ProgramGolden Key was launched in 24 Apr 2014 in Nanjing China 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 standard prophylaxis under the conditions of routine practice
But after the Recombinant Human Coagulation Factor VIII for injection ADVATE Patient Assistance ProgramGolden Key was launched in 24 Apr 2014 in Nanjing China 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 standard prophylaxis under the conditions of routine practice
Detailed Description:
Hemophilia A is an X-linked recessive congenital bleeding disorder caused by deficient or defective coagulation factor VIII FVIII The absence or severe reduction of FVIII leads to spontaneous bleeding episodes occurring primarily in joints muscles and less commonly in soft tissues and to excessive bleeding following trauma or injury Estimations based on the WFHs annual global surveys indicate that the number of people with hemophilia in the world is approximately 400000 1 China accounts for a quarter of about 100000
Currently based on the successful pioneer work of Dr Nilsson and her colleagues in Sweden that started in the late 1950s prophylaxis is recommend as the standard of care for boys with severe haemophilia by WHO and WFH 2 The efficacy and safety of prophylaxis in preventing bleeds and arthropathy in patients with hemophilia has been confirmed in well-designed clinical studies345To keep the factor level above 1 the standard dosage for patients with severe hemophilia A is 20-40 Units kginfusion average 30 Units kg 6 every other day or three times a week This dosage has a very high consumption of factor up to 5000-6000 international unitIUkgyear 7 The high consumption of factor and cost present a major barrier to use the standard prophylaxis in many countries particularly in the developing world 8 Many families are looking forward to standard prophylaxis to reducing bleeding episodes stop or slow the deterioration of joint disease in their sons with severe hemophilia and thus improving their quality of life QoL But in China the majority of boys with severe hemophilia A cannot afford the high costs of standard prophylaxis They can only pay for on-demand treatment or low-dose prophylaxis But after the Advate Patient Assistance ProgramGolden Key was launched in 24 Apr 2014 in Nanjing China 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 standard prophylaxis under the conditions of routine practice
Currently based on the successful pioneer work of Dr Nilsson and her colleagues in Sweden that started in the late 1950s prophylaxis is recommend as the standard of care for boys with severe haemophilia by WHO and WFH 2 The efficacy and safety of prophylaxis in preventing bleeds and arthropathy in patients with hemophilia has been confirmed in well-designed clinical studies345To keep the factor level above 1 the standard dosage for patients with severe hemophilia A is 20-40 Units kginfusion average 30 Units kg 6 every other day or three times a week This dosage has a very high consumption of factor up to 5000-6000 international unitIUkgyear 7 The high consumption of factor and cost present a major barrier to use the standard prophylaxis in many countries particularly in the developing world 8 Many families are looking forward to standard prophylaxis to reducing bleeding episodes stop or slow the deterioration of joint disease in their sons with severe hemophilia and thus improving their quality of life QoL But in China the majority of boys with severe hemophilia A cannot afford the high costs of standard prophylaxis They can only pay for on-demand treatment or low-dose prophylaxis But after the Advate Patient Assistance ProgramGolden Key was launched in 24 Apr 2014 in Nanjing China 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 standard prophylaxis under the conditions of routine practice
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None