Hemophilia A or B With Inhibitors Clinical Trial
Official title:
A Phase 1,Randomized, Single-blind, Placebo-controlled, Single Dose, Dose-escalated Study to Assess the Safety, Pharmacokinetic and Pharmacodynamic Profile of Subcutaneous Administration of a Long-acting Recombinant Factor VIIa in Healthy Adult Males
Verified date | September 2019 |
Source | OPKO Health, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
A Phase 1, randomized, single-blind, placebo-controlled, single dose, dose-escalation study to assess the safety, pharmacokinetic and pharmacodynamic profile of subcutaneous administration of a long-acting recombinant factor VIIa (MOD-5014) in healthy adult males.
Status | Completed |
Enrollment | 32 |
Est. completion date | February 21, 2018 |
Est. primary completion date | February 21, 2018 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | Male |
Age group | 18 Years to 50 Years |
Eligibility |
Inclusion Criteria: 1. Men, 18-50 years of age, inclusive, at the screening visit. 2. Subjects must provide written informed consent prior to participating in the study. 3. Considered healthy based on medical history, physical examination and clinical laboratory results. 4. Body Mass Index (BMI) 19.0-30.0 kg/m2 and total body weight >50 Kg. 5. Fertile men must agree to use a barrier contraceptive (condom) for 30 days post-dosing and are restricted from donating sperm for 30 days after dosing. Subjects with a vasectomy performed more than 6 months prior to treatment are also acceptable. 6. Supine blood pressure and heart rate within normal limits (systolic 90-140 mmHg; diastolic 50-90 mmHg, heart rate 45-100 beats per minute). No evidence of orthostatic hypotension. 7. Triglyceride = 200 mg/dl 8. ECG with no clinically significant abnormalities recorded at Screening visit and on dosing day (before drug administration): PR interval within 120 and 210 ms, QRS interval < 120 ms, and QTc interval 450 ms. 9. Negative human immunodeficiency virus (HIV), hepatitis B or hepatitis C serology tests at screening. 10. Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures. 11. Non-smoking and no use of any tobacco or nicotine product by declaration for a period for at least 6 month prior to screening period. Exclusion Criteria: 1. Family history of blood clots. 2. Have had, within one month prior to study drug administration, a major surgical procedure (e.g. orthopedic, abdominal) or have an elective surgery planned within the study period. 3. Any history of arterial and/or venous thromboembolic events (such as myocardial infarction, ischemic strokes, transient ischemic attacks, deep venous thrombosis or pulmonary embolism). 4. History or current drug/alcohol abuse (excluding use of medicinal cannabis for pain management). History of regular alcohol consumption exceeding - 14 drinks/week for men (1 drink = 5 ounces [150 mL] of wine or 12 ounces [360 mL] of beer or 1.5 ounces [45 mL] of hard liquor) within 6 months of screening. Positive urine drug of abuse (DoA) in screening and on admission. Positive breath alcohol test on admission. 5. Known allergy to any drug. Known allergy or hypersensitivity to any of the test compounds or materials or contraindication to test product. 6. Use of any prescription or over-the-counter (OTC) medications, including vitamins and herbal or dietary supplements within 14 days prior to dosing. Paracetamol for symptomatic relief of pain is allowed until 24 hours prior to the study drug administration. 7. Subjects who have received any vaccines within 4 weeks prior to study drug administration. 8. Participation in another clinical trial within 30 days. |
Country | Name | City | State |
---|---|---|---|
Israel | TASMC | Tel-Aviv |
Lead Sponsor | Collaborator |
---|---|
OPKO Health, Inc. |
Israel,
Bysted BV, Scharling B, Møller T, Hansen BL. A randomized, double-blind trial demonstrating bioequivalence of the current recombinant activated factor VII formulation and a new robust 25 degrees C stable formulation. Haemophilia. 2007 Sep;13(5):527-32. — View Citation
Fares FA, Suganuma N, Nishimori K, LaPolt PS, Hsueh AJ, Boime I. Design of a long-acting follitropin agonist by fusing the C-terminal sequence of the chorionic gonadotropin beta subunit to the follitropin beta subunit. Proc Natl Acad Sci U S A. 1992 May 1 — View Citation
Fridberg MJ, Hedner U, Roberts HR, Erhardtsen E. A study of the pharmacokinetics and safety of recombinant activated factor VII in healthy Caucasian and Japanese subjects. Blood Coagul Fibrinolysis. 2005 Jun;16(4):259-66. — View Citation
Klitgaard T, Nielsen TG. Overview of the human pharmacokinetics of recombinant activated factor VII. Br J Clin Pharmacol. 2008 Jan;65(1):3-11. Epub 2007 Oct 24. — View Citation
Møss J, Scharling B, Ezban M, Møller Sørensen T. Evaluation of the safety and pharmacokinetics of a fast-acting recombinant FVIIa analogue, NN1731, in healthy male subjects. J Thromb Haemost. 2009 Feb;7(2):299-305. doi: 10.1111/j.1538-7836.2008.03253.x. E — View Citation
Tanaka KA, Key NS, Levy JH. Blood coagulation: hemostasis and thrombin regulation. Anesth Analg. 2009 May;108(5):1433-46. doi: 10.1213/ane.0b013e31819bcc9c. Review. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), Immunogenicity, laboratory results, vital signs and injection site reactions | within 30 days of injection | ||
Secondary | Cmax of MOD-5014 | within 30 days of injection | ||
Secondary | Tmax of MOD-5014 | within 30 days of injection | ||
Secondary | AUC(0-t) of MOD-5014 | within 30 days of injection | ||
Secondary | AUC(inf) of MOD-5014 | within 30 days of injection | ||
Secondary | T(½) of MOD-5014 | within 30 days of injection | ||
Secondary | Clearance of MOD-5014 | within 30 days of injection |
Status | Clinical Trial | Phase | |
---|---|---|---|
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