A clinical study with a Factor VIIa (Recombinant) to evaluate its efficacy, its safety and its behaviour in human blood in pediatric patients from birth to less than 12 years of age with Hemophilia A or B with inhibitors to factor VIII or IX

Phase 1

• Conditions: Congenital Hemophilia A or B patients with inhibitors to Factor VIII or Factor IX; MedDRA version: 19.1Level: PTClassification code 10056492Term: Haemophilia A with anti factor VIIISystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 19.1Level: PTClassification code 10056494Term: Haemophilia B with anti factor IXSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2015-000958-38-CZ
• Lead Sponsor: FB USA, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-11-20
• Last Update Posted: 21 August 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 24

Inclusion Criteria

1. be male with a diagnosis of congenital hemophilia A or B of any severity
2. have one of the following:
a. a positive inhibitor test BU =5, OR
b. a BU<5 but expected to have a high anamnestic response to FVIII or FIX, as demonstrated from the patient’s medical history, precluding the use of factor VIII or IX products to treat bleeding episodes, OR
c. a BU<5 but expected to be refractory to increased dosing of FVIII or FIX, as demonstrated from the patient’s medical history, precluding the use of factor VIII or IX products to treat bleeding episodes
3. be aged from birth to <12 years old
4. have experienced at least 3 bleeding episodes of any severity in the past 6 months; if < 6 months old, have experienced at least 3 bleeding episodes since birth
5. parents or legal guardians must be capable of understanding and willing to comply with the conditions of the protocol
6. parents or legal guardians must have read, understood and provided written informed consent
Are the trial subjects under 18? yes
Number of subjects for this age range: 24
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

1. have any coagulation disorder other than hemophilia A or B
2. be immunosuppressed (i.e., the patient should not be receiving systemic immunosuppressive medication, CD4 counts at screening should be >200/µl)
3. have a known allergy or hypersensitivity to rabbits
4. have platelet count <100,000/mL
5. have had a major surgical procedure (e.g. orthopedic, abdominal) within 1 month prior to first administration of the study drug in this study
6. have received an investigational drug within 30 days of the first study drug administration, or is expected to receive such drug during participation in this study
7. have a clinically relevant hepatic (AST and/or ALT >3 times ULN) and/or renal impairment (creatinine >2 times ULN)
8. have an active malignancy (those with non-melanoma skin cancer are allowed)
9. have any life-threatening disease or other disease or condition which, in the investigator’s judgment, could imply a potential hazard to the patient, or interfere with the trial participation or trial outcome (e.g., a history of non-responsiveness to bypassing products or thromboembolic disease)
10. known or suspected hypersensitivity to the active substance or to any of its excipients

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified