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 therapy; MedDRA version: 14.1Level: LLTClassification code 10060612Term: Hemophilia ASystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2011-000410-18-PL
• Lead Sponsor: Baxter Innovations GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-12-28
• Last Update Posted: 12 November 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 100

Inclusion Criteria

•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

Exclusion Criteria

Subjects who meet any of the following criteria are not eligible for this study:
•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