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

• Status: Terminated
• Trial end date: 2011-12-28
• Target enrollment: 0
• Participant gender: Male

Summary

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Baxalta now part of Shire; Baxalta US Inc.
• Treatments: Lipoprotein-associated coagulation inhibitor

Criteria

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.