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

Baxalta

Status and phase: Terminated; Phase 1

Conditions: Hemophilia

Treatments: Drug: ARC19499; Drug: placebo control

Study type: Interventional

Funder types: Industry

Identifiers

NCT01191372

271101 (Other Identifier)

2010-020373-17 (EudraCT Number)

ARC19499-001

Details and patient eligibility

About

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.

Enrollment: 17 patients

Sex: Male

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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17 participants in 4 patient groups, including a placebo group

saline for injection

Placebo Comparator group

Treatment:

Drug: placebo control

ARC19499 Low Dose

Experimental group

Treatment:

Drug: ARC19499

ARC19499 Mid Dose

Experimental group

Treatment:

Drug: ARC19499

ARC19499 High Dose

Experimental group

Treatment:

Drug: ARC19499