An interventional study of ADVATE in Hemophilia A, sponsored by The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School. Status unknown. Open to participants aged 2 Years to 18 Years. Per ClinicalTrials.gov, last updated 2014-11-04.
Sponsored by The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School · Not applicable, Interventional, and Treatment
Based on the successful pioneer work of Dr Nilsson and her colleagues in Sweden that started in the late 1950's, 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 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.
But after the Recombinant Human Coagulation Factor VIII for injection (ADVATE) Patient Assistance Program(Golden 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.
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 WFH's annual global surveys indicate that the number of people with hemophilia in the world is approximately 400,000. [1] China accounts for a quarter of about 100,000.
Currently, based on the successful pioneer work of Dr Nilsson and her colleagues in Sweden that started in the late 1950's, 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 studies.[3,4,5]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) [6] 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. [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 Program(Golden 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.
866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.
This study's planned enrollment of 15 is below the median of 28 across 512 interventional studies indexed under Hemophilia A.
Browse Hemophilia A studies →The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School is the lead sponsor of 242 studies on the registry; 144 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
This study is a prospective, open-label, interventional, multicenter study in a total of 15 PTPs with hemophilia A (FVIII≤2 %).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 2-3 times one week with 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: 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 2-3 times one week with ADVATE for 1 year.
Also known as: Recombinant Human Coagulation Factor VIII for injection
Annual Bleed Rate (ABR)
Every 3 months, evaluate the joint bleeding episode and any other bleeding episodes. After 1 year later, sum up all the bleeding and get the ABR.
Time frame: 3 months up to 1 year
incidence of new target joints
Incidence of new target joints.
Time frame: 3 months
joint health of Pettersson score
Status of joint health by X-ray using Pettersson scale.
Time frame: 1 year
magnetic resonance imaging scoring of joint
Status of joint health by magnetic resonance imaging scoring system.
Time frame: 6 months
Hemophilia Joint Health Score (HJHS) of joint
Status of joint health using the Hemophilia Joint Health Score (HJHS)
Time frame: 3 months
Number of Recombinant Human Coagulation Factor VIII for injection (ADAVTE) units
Number of ADVATE infusions required for bleed cessation and Number of ADAVTE units required for bleed cessation
Time frame: 3 months
Qol assessment
QoL assessment by The Canadian Hemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT)
Time frame: 3 months
Inhibitor rate
Inhibitor rate monitoring: inhibitor will be tested at entry of study, every 3 months and at end of study.
Time frame: 3 months up to 1 year
No study locations are listed for this record.
This study is status unknown, as verified in Oct 2014. You cannot join it, but the record below documents what was studied.
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The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School