A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A
Status: Recruiting
Phase:
Diagnosis: Pediatric Hematology/Blood Related
NCT ID: NCT01233258
(View complete trial on ClinicalTrials.gov)
DFCI Protocol ID:
The objective of the trial is to demonstrate that 2-3 times per week prophylaxis therapy with BAY81-8973 is superior to on-demand therapy with BAY81-8973 in patients with severe Hemophilia A. The hypothesis is that prophylaxis will result in fewer bleeds than on-demand treatment.
Conducting Institutions:
Children's Hospital Boston, Dana-Farber Cancer Institute
Overall PI:
Kapil Saxena, MD,
Children's Hospital Boston
Site-responsible Investigators:
Contacts:
Dana-Farber Cancer Institute:
Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP,
ctip@partners.org
Eligibility Criteria
Inclusion Criteria:
- Male, aged 12 to 65 years
- Severe hemophilia A
- History of more than 150 exposure days (ED) with clotting factor concentrates
- Currently receiving episodic treatment with FVIII; no regular prophylaxis for more
than 6 consecutive months in the past 5 years
- No current Factor VIII inhibitor or history of inhibitor
- Willing to use electronic patient diary
Exclusion Criteria:
- Presence of another bleeding disease that is different from hemophilia A
- Thrombocytopenia
- Abnormal renal function
- Presence of active liver disease
- Known hypersensitivity to FVIII