A Phase 3b Clinical Study To Assess Whether Regular Administration Of Advate In The Absence Of Immunological Danger Signals Reduces The Incidence Rate Of Inhibitors In Previously Untreated Patients With Hemophilia A

Phase 3

Completed

• Conditions: Coagulation Factor VIII deficiency / Blood disease; 10018849

• Registration Number: NL-OMON35535
• Lead Sponsor: Baxter

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 6 May 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 6

Inclusion Criteria

1. 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
2. Subjects < 1 year of age
3. 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.
4. 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 no more than 2 EDs of FVIII treatment were required.
5. Adequate venous access (without need for CVAD-placement) as determined by the physician
6. Written informed consent from legally authorized representive(s)

Exclusion Criteria

1. Life-threatening conditions (intracranial hemorrhage, severe trauma) or requirement for surgery at the time of enrollment
2. 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 inhibitor test results)
3. Inherited or acquired hemostatic defect other than hemophilia A
4. Any clinically significant, chronic disease other than hemophilia A
5. Known hypersensitivity to ADVATE or any of its constituents
6. Any planned elective surgery that cannot be postponed until after the first 20 EDs
7. Participation in the Hemophilia Inhibitor PUP Study (HIPS)
8. Application of red blood cell, platelet, or leukocyte concentrates, or plasma
9. Administration of any medication affecting coagulation or platelet function
10. Systemic administration of any immunomodulatory drug (eg, chemotherapy, intravenous glucocorticoids)
11. Participation in another clinical study involving an IP or device within 30 days prior to study enrollment or during the course of this study.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Incidence of inhibitor formation in severe and moderately severe hemophilia A<br /><br>(FVIII <= 2%) within the first 50 EDs to ADVATE (prior exposure to FVIII up to a<br /><br>maximum of 3 EDs but maximum 2 exposures per event - to any FVIII concentrate<br /><br>are allowed, and infusions for bleed management will be included in the 50-ED<br /><br>calculation)</p><br>