A clinical study with a Factor VIIa (Recombinant) to evaluate its efficacy, its safety and its behaviour in human blood in patients with Hemophilia A or B with inhibitors to factor VIII or IX

Phase 1

• Conditions: Congenital haemophilia A or B patients with inhitors to Factor VIII or Factor IX; MedDRA version: 17.0Level: LLTClassification code 10018938Term: Haemophilia A (Factor VIII)System Organ Class: 100000004850; MedDRA version: 17.0Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2013-004779-11-RO
• Lead Sponsor: rEVO Biologics, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-06-02
• Study Completion: 2015-07-31
• Last Update Posted: 17 January 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 27

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 (as confirmed at screening by the institutional lab), OR
b. a BU <5 but expected to have a high anamnestic response to FVIII or FIX, as demonstrated from the subject's medical history, precluding the use of the factor VIII or IX products to treat bleedings, OR
c. a BU <5 but expected to be refractory to increased dosing of FVIII or FIX, as demonstrated from the subject's medical history, precluding the use of the factor VIII or IX products to treat bleedings
3. be 12 years or older, up to and including 75 years of age
4. have at least 3 bleeding episodes of any severity in the past 6 months
5. be capable of understanding and willing to comply with the conditions of the protocol
6. have read, understood and provided written informed consent (patient and/or parent(s)/legal guardian(s) if <18 years age)
Are the trial subjects under 18? yes
Number of subjects for this age range: 6
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 17
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

1. have any coagulation disorder other than hemophilia A or B
2. be immuno-suppressed (i.e., the patient should not be receiving systemic immunosuppressive medication, CD4 counts at screening should be >200/µl)
3. have known allergy or hypersensitivity to rabbits
4. have platelet count <100,000/ml
5. have had within one month prior to first administration of the study drug in this study a major surgical procedure (e.g. orthopedic, abdominal)
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 the upper limit of normal) and/or renal impairment (creatinine >2times the upper limit of normal)
8. have a history of arterial and/or venous thromboembolic events (such as myocardial infarction, ischemic strokes, transient ischemic attacks, deep venous thrombosis or pulmonary embolism) within 2 years prior to first dose of study drug, or current New York Heart Association (NYHA) functional classification score of stage II-IV
9. have an active malignancy (those with non-melanoma skin cancer are allowed)
10. have any life-threatening disease or other disease or condition which, according to the investigator's judgment, could imply a potential hazard to the patient, interfere with the trial participation or trial outcome (e.g., a history of non-responsiveness to bypassing products)

Study & Design

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