A Study of the Safety and Tolerability of FEIBA Reconstituted in Regular or 50% Reduced Volume and of Faster Infusion Rates in Patients with Hemophilia A or B with Inhibitors

Phase 1

Conditions: Hemophilia A or B with inhibitors
MedDRA version: 20.0Level: LLTClassification code 10060612Term: Hemophilia ASystem Organ Class: 100000004850
MedDRA version: 20.0Level: LLTClassification code 10060614Term: Hemophilia B (Factor IX)System Organ Class: 100000004850
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2015-005781-39-RO

Lead Sponsor: Baxalta Innovations GmbH

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 32

Inclusion Criteria

1. = 18 to = 65 years old at the time of screening
2. Hemophilia A or B of any severity, with a documented = 3 months history of inhibitors (= 0.6 BU) requiring the use of bypassing agents (FEIBA or rFVIIa) prior to screening. Inhibitor level will be tested at screening if no documented history is available.
3. Hepatitis C virus (HCV) negative, either by antibody testing or polymerase chain reaction (PCR); or HCV positive with stable liver disease
4. Human immune deficiency virus (HIV) negative; or HIV positive with stable disease and CD4 count = 200 cell/mm3 at screening
5. Adequate venous access
6. Willing and able to comply with the requirements of the protocol
7. If a female of childbearing potential, must have a negative blood pregnancy test and agrees to employ adequate birth control measures for the
duration of the study, such as:
a. Abstain from sexual intercourse
b. Use a reliable method of contraception (contraception such as an intrauterine device, barrier method [e.g., diaphragm or sponge; female condom not permitted] with spermicide, oral contraceptive, injectable progesterone, sub dermal implant), and have their male partner use a condom
8. If female of non-childbearing potential, confirmed at screening by fulfilling 1 of the following criteria:
a. Postmenopausal, defined as amenorrhea for at least 12 months following cessation of all exogenous hormonal treatments and with follicle-stimulating hormone levels within the laboratory-defined postmenopausal range or postmenopausal with amenorrhea for at least 24 months and on
hormonal replacement therapy
b. Documentation of irreversible surgical sterilization by hysterectomy, bilateral oophorectomy, bilateral tubal ligation (with no subsequent
pregnancy at least 1 year from bilateral tubal ligation), or bilateral salpingectomy
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 32
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Known hypersensitivity to FEIBA or any of its components
2. Advanced liver disease (e.g., liver biopsy confirmed diagnosis of cirrhosis, portal vein hypertension, ascites, prothrombin time [PT] 5 seconds above upper limit of
normal)
3. Planned elective surgery during participation in this study (excluding minor procedures that will not need preventative bleeding treatments, such as exchanges of peripherally inserted central catheters)
4. Platelet count < 100,000/µL
5. Taking Emicizumab (Hemlibra) for bleed prevention
6. Clinical or laboratory evidence of disseminated intravascular coagulation based on medical history
7. Prior history or evidence of thromboembolic event: acute myocardial infarction, deep vein thrombosis, pulmonary embolism, etc.
8. Diagnosis of advanced atherosclerosis, malignancy, and/or other diseases that may increase the subject’s risk of thromboembolic complications
9. Taking any immunomodulating drug (e.g., corticosteroid agents at a dose equivalent to hydrocortisone > 10 mg/day, or a-interferon) within 30 days prior to enrollment except anti-retroviral chemotherapy.
10. Herbal supplements that contain anti-platelet activity
11. Subject has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study
12. Family member or employee of the investigator
13. Clinically significant medical, psychiatric or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject safety or compliance

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

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