First-in-Human and Proof-of-Mechanism Study of ARC19499 Administered to Hemophilia Patients

Overview

The purpose of this study is to examine the safety, tolerability and the way the body handles various single and multiple doses of ARC19499 in patients with hemophilia.

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: December 2011

Interventions

  • Drug: placebo control
    • sterile saline for injection
  • Drug: ARC19499
    • Anti-tissue factor pathway inhibitor (TFPI) aptamer

Arms, Groups and Cohorts

  • Placebo Comparator: saline for injection
  • Experimental: ARC19499 Low Dose
  • Experimental: ARC19499 Mid Dose
  • Experimental: ARC19499 High Dose

Clinical Trial Outcome Measures

Primary Measures

  • Pharmacokinetics (PK) of ARC19499
    • Time Frame: 2 weeks
    • The PK profile of ARC19499 administered by single and multiple subcutaneous injections will be characterized. The bioavailability of subcutaneously injected ARC19499 relative to that of intravenously infused ARC19499 will be determined.

Secondary Measures

  • Coagulation system pharmacodynamic (PD) effects of ARC19499.
    • Time Frame: 2 weeks
    • The PD profile of ARC19499 with respect to the kinetics of thrombin generation and clot formation will be characterized.

Participating in This Clinical Trial

Inclusion Criteria

Adult male patients ≥18 to ≤75 years of age.

  • Hemophilia of any type or severity. – Patients who are negative for hepatitis B surface antigen (HBsAg), and human immunodeficiency virus (HIV) I and II antibody tests at screening. – Male patients who, with their partners, are willing to use 2 effective, methods of contraception (i.e., for both self and partner) throughout the study and for at least 3 months after discontinuation of study drug treatment. – All patients must be capable of understanding and complying with the protocol and must have signed the informed consent document. Exclusion Criteria:
  • Female patients; – If on a prophylactic coagulation factor concentrate regimen, inability or unwillingness to discontinue prophylaxis during participation in this study. – Existence of other co-existing bleeding disorder (e.g., von Willebrand Disease). – Medical history of venous or arterial thromboembolism. – Scheduled for elective surgical procedure during the conduct of this study. – Use of an investigational drug within 30 days of study entry. – Transaminase values > 3 x upper limit of normal (ULN) at time of screening. – Haemoglobin <12.0 g/dL. – Participants who, in the opinion of the Investigator, have a significant infection or known inflammatory process on screening. – Participants who have a clinically relevant history or presence of respiratory, gastrointestinal, renal, hepatic, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, connective tissue diseases or disorders. – Any medical condition the investigator believes would place the patient at increased risk as a result of participation in the study e.g. history of thromboembolic disease or stroke. – Any medication the investigator considers may increase the risk of adverse effects during the study.
  • Gender Eligibility: Male

    Minimum Age: 18 Years

    Maximum Age: 75 Years

    Are Healthy Volunteers Accepted: No

    Investigator Details

    • Lead Sponsor
      • Baxalta now part of Shire
    • Provider of Information About this Clinical Study
      • Sponsor
    • Overall Official(s)
      • Wing-Yen Wong, MD, Study Director, Baxter Healthcare Corporation

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