Low dose factor VIII prophylaxis is practiced around the world. Role of standard dose Emicizumab prophylaxis is well established. Emicizumab is an expensive drug. Standard dose Emicizumab prophylaxis is very expensive for hemophilia A patients and troublesome for government to ensure continuous supply. This study intends to compare low dose Factor VIII prophylaxis with low dose Emicizumab prophylaxis and compare whether low dose Emicizumab is as effective as low dose Factor VIII prophylaxis. So, it is possible to continue prophylaxis program in hemophilia A patients with a cost-effective way in our country without risking the patient health. Moreover, Emicizumab prophylaxis reduces the chance of developing inhibitor to Factor VIII and it is convenient for administration due to less frequent and subcutaneous administration. 20 severe hemophilia A patients will be selected randomly from interested patients for this study. 6 patients with inhibitor will be randomized in Low dose Emicizumab with Inhibitor group (I) and rest 14 will be randomized by block randomization in Low dose Emicizumab (without inhibitor-WI) and low dose Factor VIII group at 4:10 ratio. Initial loading dose will be given and the participants will be followed up for 6 months. At the end of the study Annualized bleeding rate (ABR), Annualized joint bleeding rate (AJBR), Annualized spontaneous bleeding rate (ASBR), APTT will be compared among the groups. Inhibitor to factor VIII will also be evaluated after six months in Factor VIII group.
Severe hemophilia A patient with or without inhibitor to factor VIII.
1. Severe hemophilia A patient not willing to participate.
Parallel
Emicizumab Injection
DrugEmicizumab 0.8-1.5mg/kg weekly for 4 weeks as loading dose. Then 0.8-1.5mg/kg 4-weekly for 24 weeks will be used in Emicizumab group-I and Emicizumab Group- WI
Factor VIII (FVIII)
Drug10-15 unit/Kg thrice weekly (for conventional half-life products) or twice weekly (for extended half-life products) for 28 weeks. Inj. Factor VIII is available in 250U, 500U, 750U \& 1,000 IU vail sizes. Dose will be round up to full nearest full vail strength.
Mean of Annualized bleeding rate (ABR) among the study groups will be compared.
ABR will be calculated as the number of reported bleeding events divided by 06 (the number of months in the reporting time window) and multiplied by 12. e.g. if a participant total bleeding event are 12 in 6 months, ABR of this patients is 12/6\*12=24
Mean of Annualized joint bleeding rate (AJBR) among the study groups will be compared.
AJBR will be calculated as the number of reported joint bleeding events divided by 06 (the number of months in the reporting time window) and multiplied by 12. e.g. if a participant Joint Bleeding event is 6 in 6 months than AJBR will be 6/6\*12=12.
Mean of Annualized spontaneous bleeding rate (ASBR) among the study groups will be compared.
ASBR will be calculated as the number of reported spontaneous bleeding events divided by 06 (the number of months in the reporting time window) and multiplied by 12. e.g. if a participant has total 4 spontaneous bleeding event in six months than ASBR will be 4/6\*12= 7.99
Mean of APTT among the groups will be compared.
APTT
Inhibitor to Factor VIII-
Check for development of inhibitor to Factor VIII in Factor VIII group and emicizumab group without inhibitor.
Click on the lead sponsor to view all of their trials.
This trial is not recruiting at the moment. You can still explore other options:
