Clinical study to find out whether regular infusions of ADVATE in the absence of immunological danger signals (eg. tissue damage/viral or bacterial infections) reduces the possibility of inhibitor in PUPs with hemophilia A
- Conditions
- Prevention of inhibitor formation, and immune tolerance induction in patients with severe and moderately severe hemophilia A by early and low-dose prophylactic ADVATE therapyMedDRA version: 14.0Level: LLTClassification code 10060612Term: Hemophilia ASystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2011-000410-18-ES
- Lead Sponsor
- Baxter Innovations GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 100
Subjects who meet all of the following criteria are eligible for this study:
- Subjects with severe and moderately severe hemophilia A (FVIII =< 2%)
- Certain FVIII mutation types (eg, large multi-domain deletions; nonsense mutations; insertions/deletions/inversions that result in a premature stop codon; intron 22 inversions) can be used to corroborate a severe hemophilia A phenotype when laboratory assays show FVIII levels >=1% because of rounding errors or carryover effect from a previous FVIII administration; a central laboratory FVIII assay is required to confirm subject eligibility.
- Subjects < 1 year of age.
- Subjects must have =< 3 EDs to any FVIII concentrate or FVIII-containing product used for treatment of minor bleeds (bleeds requiring no more than 2 infusions per event), or for preventative or precautionary infusions following possible injury.
- Subjects with prior circumcision are allowed to enroll only if bleeding issues related to circumcision were the cause for the original diagnosis of hemophilia A and if no more than 2 EDs of FVIII were required.
- Adequate venous access (without need for central venous access device [CVAD]-placement) as determined by the physician
- Written informed consent from legally authorized representative(s)
Are the trial subjects under 18? yes
Number of subjects for this age range: 100
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Life-threatening conditions (intracranial hemorrhage, severe trauma), or requirement for surgery at the time of enrollment
- Evidence of inhibitor >= 0.6 BU in Njimegen-modified Bethesda Assay at study start (samples may be retested using lupus-insensitive inhibitor tests to reduce the number of false positive inhibitortesting for lupus anticoagulant will be done to eliminate false positives)
- Inherited or acquired hemostatic defect other than hemophilia A
- Any clinically significant, chronic disease other than hemophilia A
- Known hypersensitivity to ADVATE or any of its constituents
- Any planned elective surgery that cannot be postponed until after the first 20 EDs
- Participation in the Hemophilia Inhibitor PUP Study (HIPS)
- Application of red blood cell, platelet, or leukocyte concentrates, or plasma
- Administration of any medication affecting coagulation or platelet function
- Systemic administration of any immunomodulatory drug (eg, chemotherapy, intravenous glucocorticoids)
- Participation in another clinical study involving an investigational product (IP) or device within 30 days prior to study enrollment or during the course of this study
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method